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Current Research Projects

Mechanisms and Rehabilitation of Cerebellar Ataxia
Principal Investigator:
Bastian, Amy
Sponsored by the National Institutes of Health -- 2R01HD040289. Cerebellar damage disrupts movement coordination. Actions ranging from walking to reaching become inaccurate and difficult to control, resulting in poor functional movements. Therapeutic approaches for cerebellar motor disorders, or "...
Human Locomotor Plasticity in Health and Disease
Principal Investigator:
Bastian, Amy
Sponsored by the National Institutes of Health -- 5R01HD048741-06. Walking patterns can adapt on short timescales (minutes to hours) to temporary conditions, such as icy sidewalks. They can also adapt on long timescales (months to years) to permanent conditions produced by growth or damage. Walking...
The Development of a Reading Intervention for Deaf and Hard-of-Hearing Students
Principal Investigator:
Beetar, John
The specific aim of this project is to complete the development of a computer software program of reading instruction, Sign 'n' Read, for deaf and hard-of-hearing students. Presently in development as a prototype, Sign 'n' Read will consist of two modules, a sight-word module and a vocabulary...
Maternal Child Health Careers/Research Initiatives -- Undergraduate Program
Principal Investigator:
Belcher, Harolyn
Sponsored by DHHS/CDC -- 1U50MN000025. Healthy People 2020 highlights the importance of eliminating health disparities and increasing the quality and years of healthy life free of preventable disease, disability, and injury. Current scientific evidence overwhelmingly documents the...
Kennedy Krieger Center for Child and Family Traumatic Stress - Integrated Trauma Approaches
Principal Investigator:
Belcher, Harolyn
The goals of the Kennedy Krieger Centet for Child and Family Traumatic Stress Trauma Intervention Program (TIP) are to: Evaluate the feasibility and effectiveness of evidence-based treatment protocols, endorsed by the National Child Traumatic Stress Network, that specifically address...
Relationship of MRI to ASIA Impairment Scale in Chronic Spinal Cord Injury
Principal Investigator:
Belegu, Visar
Structural & functional MRI studies of the central & peripheral nervous system in healthy children & children with spinal cord injury at 1.5 & 3.0 T Development of imaging correlates of the integrity of spinal cord white matter is needed for optimizing design of clinical trials...
A 12 Week Double Blind Placebo Controlled Clinical Trial to Evaluate the Safety and Efficacy of Rivastigimine in Children (ages 10-17) with Down Syndrome
Principal Investigator:
Capone, George
Individuals with Down syndrome (DS) exhibit a notable cholinergic deficiency by the 4th decade of life. It is unclear how early in life presynaptic cholinergic deterioration begins or how it evolves in subsequent decades. Acetylcholinesterase inhibitors (AchEIs) may improve cognitive and language...
Intellectual and Developmental Disabilities Research Centers 2008
Principal Investigator:
Cataldo, Michael
This application for continued support of Years 21 through 25 of the Mental Retardation and Developmental Disabilities Research Center (MRDDRC) at the Kennedy Krieger Institute and Johns Hopkins University is submitted in response to RFA-HD-07-012. The MRDDRC consists of the Administrative Core (...
Developing a Means of Comparing Expenditures at Three Models of Nonpublic Special Education Services at a Private Special Education School in Baltimore, Maryland
Principal Investigator:
Church, Robin
Currently, there is no uniform means of determining the true cost of nonpublic special education in Maryland. Today, tuition is used as “the cost” of providing these programs. The proposed project will identify a formula that nonpublic schools and Local Education Agencies may use to determine per...
Anticonvulsants, Ischemic Seizures and Regeneration in the Immature Brain
Principal Investigator:
Comi, Anne
Neonatal stroke affects one in 4000 term births and frequently results in cognitive impairments. Neonatal strokes present with seizures, and anticonvulsants (usually phenobarbital) are administered for months afterward. Our central hypothesis is that impaired hippocampal neurogenesis after neonatal...
EEG-Based Assessment of Functional Connectivity in Autism
Principal Investigator:
Ewen, Joshua
Sponsored by the National Institutes of Health -- 1K23NS073626-01. There are substantial data to suggest that altered cerebral connectivity is a key aspect of the neurobiology of autism. Nevertheless, the role of altered cerebral connectivity in the production of the core impairments of autism is...
Mentored Clinical Scientist Research Career Development Award
Principal Investigator:
Fatemi, S. Ali
The proposed training will prepare a young pediatric neurology faculty member for a career as an independent investigator in the field of translational neuroscience research, focusing on perinatal disorders of white matter development. This research will be performed at the Hugo Moser Research...
Comprehensive Long-Term Evaluation of the Gunn Rat Kernicterus Model
Principal Investigator:
Fatemi, S. Ali
The proposed project is the initial phase of a long-term endeavor to develop therapies that will help patients with dyskinetic cerebral palsy related to mitochondrial dysfunction and a Kernicterus-like phenotype. We have evidence that the combination of a genetic predisposition affecting...
Study of White Matter Development in a Rodent Model of Perinatal Brain Injury
Principal Investigator:
Fatemi, S. Ali
This is a Mentored Clinical Scientist Research Career Development Award allowing Dr. Fatemi to develop a career as an independent investigator in the field of translational neuroscience research, focusing on perinatal disorders of white matter development. This research is performed at the Hugo...
An adaptive, phase IIB/III, double-blind, randomized, placebo-controlled, multi-center study of the safety and efficacy of NaBen® (sodium benzoate), a d-amino acid oxidase inhibitor, as an add-on treatment for schizophrenia in adolescents
Principal Investigator:
Findling, Robert
Phase IIb Objectives: The primary objective of the Phase IIb portion of this study is to evaluate NaBen® (sodium benzoate) (1000 mg/day) safety and effectiveness and to determine the final sample size needed in order to proceed with the Phase III part of this trial. The secondary objective of this...
Validation Study of the Aggression Diary
Principal Investigator:
Findling, Robert
The primary objective of this study is to conduct psychometric tests on the electronic observer- reported (eObsRO) Aggression Diary to evaluate whether the diary is a valid and reliable measure for assessing the benefit of SPN-810 (Molindone hydrochloride extended-release tablets) for the treatment...
Phase 2 of Growth Hormone for Treatment of Albright Hereditary Osteodystrophy
Principal Investigator:
Germain-Lee, Emily
Sponsored by the Food and Drug Administration -- 5R01FD003409-02. Albright hereditary osteodystrophy (AHO) is a rare genetic disorder caused by heterozygous inactivating mutations in the gene encoding the chain of Gs. Among other abnormalities, it is associated with short stature and obesity....
Osteogenesis Imperfecta Clinic
Principal Investigator:
Germain-Lee, Emily
Sponsored by the Harry Headley Charitable & Research Foundation. Funding for an independent clinician/researcher to work with Dr. Jay Shapiro in the OI Clinic.
Retrospective Chart Review of Growth in Osteogenesis Imperfecta
Principal Investigator:
Germain-Lee, Emily
The purpose of this research is to assess the growth parameters(height and weight) in osteogenesis imperfecta during infancy, childhood, and adolescence with the ultimate goal of determining whether there are key points in time during which growth is impaired. We will also tabulate adult heights...
Reward System in Autism
Principal Investigator:
Goldberg, Melissa
It is a core feature of autism that social interactions appear to be far less rewarding for autistic children compared to typically developing children. Based on this observation, one would predict that children with autism would show different behavioral responses to reinforcers involving social...
An evaluation of differential reinforcement on appropriate toy skills
Principal Investigator:
Gonzalez, Melissa
The purpose of the current study will be to increase the play skills for six children with developmental disabilities, who display limited interest in toys in a free operant setting. It is hypothesized that differential reinforcement of appropriate play behaviors combined with fading will increase...
Delineating subtypes of self-injurious behavior maintained by automatic reinforcement
Principal Investigator:
Hagopian, Louis
Self-injurious behavior (SIB) is one of the most serious problems experienced by individuals with intellectual disabilities. The standard of care for the assessment of SIB involves conducting a "functional analysis." This assessment procedure involves observing the individual under several analog...
Translational Analyses of Chronic Aberrant Behavior Across the Lifespan
Principal Investigator:
Hagopian, Louis
The goal of the proposed line of research is to gain a better understanding of the processes that evoke and maintain severe problem behavior (i.e., chronic aberrant behavior) in persons with intellectual and developmental disabilities to improve treatment approaches and outcomes. Despite the...
Behavioral Economics in MR/DD and Behavior Disorders (2)
Principal Investigator:
Hagopian, Louis
Assessments of severe behavior disorders of children with intellectual and developmental disabilities (IDD) have revealed that behavior problems are often maintained by their functional outcomes (or reinforcers). For example, children may gain caregiver attention or escape instructional demands via...
Quantitative mapping of the basal ganglia and related structures in children with dyskinetic cerebral palsy
Principal Investigator:
Hoon, Alexander
Cerebral palsy (CP) refers to a group of motor-impairment syndromes secondary to genetic and acquired disorders of early brain development.  When the underlying disorder affects the basal ganglia (BG) and cortico-striato-thalamic-cortical (CSTC) circuits- from etiologies including acute...
Neurotrophin Protection of Hypoxic Ischemic Brain Injury
Principal Investigator:
Hossain, Mir Ahamed
Perinatal hypoxia-ischemia (HI) is a major cause of life-long neurological disability such as cerebral palsy. There are few treatments available for this devastating disorder. For the next period of support we propose to study more in-depth of a novel molecular target of HI brain injury based on...
Functional and Neurovascular Biomarkers for Huntington’s disease using MRI at 7T
Principal Investigator:
Hua, Jun
Oxidative stress and metabolic disturbances in the brain are believed to be involved in HD pathogenesis. Functional and neurovascular brain changes may represent altered neuronal physiology and metabolism.  These changes may be detectable prior to anatomical changes.  Early detection of...
Research Training in Rehabilitation for Brain Injury and Neurological Disability
Principal Investigator:
Johnston, Michael
This is the first resubmission of an application for renewal of an Institutional National Research Service Award for a successful postdoctoral training program in medical rehabilitation research that focuses on brain injury and neurological disability. The PIs propose to train 4 postdoctoral level...
Cholesterol Homeostasis in Malignant Glioma
Principal Investigator:
Kolar, Elizabeth
The Watkins Lab has observed that when U87 human malignant glioma cells are deficient for a particular acyl- CoA synthetase, ACSVL3, the cells had significantly lower cholesterol levels than did wild-type U87 cells. Depleting U87 cells of ACSVL3 decreased their malignant growth properties in vitro...
Effects of Self-Generated Experiences on Social Cognitive Development in Young Children with Autism
Principal Investigator:
Landa, Rebecca
Sponsored by Autism Speaks -- 7746. This study will test whether a novel treatment for social impairment in young children with autism spectrum disorders (ASD) leads to improvement at behavioral and social information processing levels, addressing Autism Speaks' priority of developing and...
Multisite RCT of Early Intervention for Spoken Communication in Autism
Principal Investigator:
Landa, Rebecca
While significant progress has been made toward identifying effective interventions for preschool-age children with autism (National Research Council, 2001), few scientifically rigorous studies have compared active ingredients of these interventions or examined outcomes focused on core deficits. To...
Autism: Social and Communication Predictors in Siblings
Principal Investigator:
Landa, Rebecca
This is a longitudinal study of Autism Spectrum Disorders (ASD). Six groups will be assessed longitudinally. Group 1: siblings of probands with autism; (Group 2) late talkers having no family history of autism; (Group 3) typically-developing controls; (Group 4) premature controls (for the motor...
MCH Research - Parent-Mediated Vs. Center-Based Intervention for Toddlers with ASD: An RCT
Principal Investigator:
Landa, Rebecca
An ever increasing number of toddlers are identified as being at high risk for ASD. This translates into an increasing demand for Part C Early Intervention services. The importance of intervention for toddlers with ASD is considerable, given the role that early experience plays in the establishment...
Development of a Social and Communication Intervention for Preschoolers with Autism
Principal Investigator:
Landa, Rebecca
RFA topic and goal:  Autism Spectrum Disorders, Development and Innovation Goal Two Purpose:  Develop and document feasibility and promise of a social and communication curriculum, and guidelines for intervention materials, activities and instructional strategies for preschoolers with...
Brain Cancer Stem Cell Reprogramming by C-MET
Principal Investigator:
Laterra, John
Glioblastoma (GBM) is an almost universally fatal brain tumor with an average median survival of only ~15 months. New effective therapies are desperately needed. Small subpopulations of malignant cells with stem cell-like properties exist in GBM. These neoplastic stem-like cells (GBM SCs) are...
Suppression of Glioblastoma Stem Cells by Kruppel-Like Factor 9
Principal Investigator:
Laterra, John
Glioblastoma stem-like cells (or cancer stem cells, CSCs) maintain a capacity for multi-lineage differentiation and efficiently propagate tumor xenografts that accurately recapitulate the complex histopathology of clinical glioblastoma. Identifying and targeting the molecular mechanisms that...
Neuroimaging, Behavior, and Biochemical Correlates in Smith-Lemli-Opitz Syndrome (SLOS)
Principal Investigator:
Lee, Ryan
SLOS is an autosomal recessive, multiple malformation syndrome of impaired cholesterol biosynthesis resulting in decreased cholesterol and elevated concentrations of abnormal sterols in tissues and blood (Irons, 1993). Cholesterol serves important biologic functions. It is a structural component of...
Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
Principal Investigator:
Leung, Doris
This study, supported by the FSH Society, will utilize magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to develop non-invasive, quantitative imaging biomarkers for facioscapulohumeral muscular dystrophy (FSHD), a genetic disorder that causes progressive skeletal muscle...
Interactive Autism Network Project
Principal Investigator:
Lipkin, Paul
The Interactive Autism Network (IAN) was established in 2006 to accelerate autism spectrum disorder (ASD) related research by creating a national online registry and longitudinal research database and by facilitating communication between the autism community and researchers. In addition, IAN...
Monitoring Prodrug Delivery in Suicide Gene Therapy Using CEST MRI
Principal Investigator:
Liu, Guanshu
To accelerate the development of nanotechnologies for drug and gene delivery, it is highly desired to construct nanoparticles with imaging capabilities so that the process of delivery and release can be monitored and quantified with a medical imaging modality. Although there have been successful...
Development of ADHD in Preschool Children: Neuroimaging and Behavioral Correlates
Principal Investigator:
Mahone, E. Mark
Attention-Deficit/Hyperactivity Disorder (ADHD) has become the most commonly diagnosed form of psychopathology in the preschool years. To address this alarming trend, the overall goal of the proposed investigation, "Development of ADHD in Preschool Children: Neuroimaging and Behavioral Correlates"...
Optic Nerve Head Synucleinopathy in Glaucoma and Function of Gamma-Synuclein
Principal Investigator:
Marsh-Armstrong, Nicholas
The broad long-term goal of this application is to uncover the mechanism of retinal ganglion cell (RGC) degeneration in glaucoma so as to be able to design rational therapeutic interventions based on neuroprotection. Based on recent data showing that glaucoma animal models develop aggregates of y-...
Transcellular Degradation of Mitochondria in the Central Nervous System
Principal Investigator:
Marsh-Armstrong, Nicholas
We recently made the surprising discovery that mitochondria from retinal ganglion cell axons are passed onto neighboring astrocytes for degradation in the optic nerve head of normal mice. We present evidence that this pathway is conserved in species ranging from frogs to primates, and that it...
Developing New Multicolor Polypeptide MR Contrast Agents for Stem Cell Tracking
Principal Investigator:
McMahon, Michael
Development of new contrast materials is important to expand the diagnostic capabilities of MR at the cellular and molecular level. In particular, providing information on cell migration, trafficking and homing would be useful for the development of cell therapies using stem cells and progenitors....
Multi-Color Exchange Transfer Imaging of Drug Delivery Nanocarriers
Principal Investigator:
McMahon, MIchael
This project focuses on the design of biodegradable contrast agents, based on peptides and heterocycles, to be used for the labeling of drug delivery vehicles such as liposomes and polymeric particles. Drugs for systemic and local administration of chemotherapy will be packaged together with these...
OIACEST Islet Cell Capsules for Immunoprotection, MR Detection, and pH Sending
Principal Investigator:
McMahon, Michael
Good glycemic control is critical in reducing the end-stage complications of Type I Diabetes Mellitus (TIDM) and, for this reason, islet transplantation (ITx) has received considerable attention in recent years, with one of the complications in the treatment involving finding an effective means to...
Motor Skill Learning in Autism
Principal Investigator:
Mostofsky, Stewart
Abnormalities on motor examination have often afforded valuable insights into developmental disorders of the brain; such abnormalities have been well documented in individuals with autism and, in fact, date back to some of the original descriptions of the disorder. Increased insight into the brain...
Anomalus Motor Physiology in ADHD
Principal Investigator:
Mostofsky, Stewart
The hypothesis of this study is that the neuromotor anomalies present in children with ADHD will be associated with deficient intracortical and interhemispheric inhibition of the motor cortex. This hypothesis is consistent with studies suggesting that the central deficit of ADHD is a failure to...
Placebo-Controlled Trial of Dextromethorphan in Rett Syndrome
Principal Investigator:
Naidu, SakkuBai
Dr. Sakkubai Naidu, Principal Investigator, is initiating a double blinded placebo controlledclinical drug trial using dextromethorphan (DM) in Rett Syndrome (RTT), at the PediatricClinical Research Unit (PCRU) of the Johns Hopkins Hospital/Kennedy Krieger Institute, that issponsored by the FDA and...
Pathogenesis of Rett Syndrome
Principal Investigator:
Naidu, SakkuBai
This is an observational study of the natural history and clinical course of Rett Syndrome (RTT), a neurodevelopmental disorder primarily affecting females, caused by mutations in the MeCP2 gene. Our hypothesis is to characterize contributors to the phenotypic variability among mutations in MeCP2...
Understanding the Brain Basis of Impaired Imitation Learning in Autism
Principal Investigator:
Nebel, Mary Beth
In a developmental disorder such as autism (ASD), brain-based learning mechanisms vital to the acquisition of motor skills may contribute to impaired development of social and communicative abilities. One problem that likely relates to both motor and social/communicative skill acquisition in ASD is...
The Role of the Transcallosal Pathway in Neuroplasticity Following Nerve Injury
Principal Investigator:
Pelled, Galit
Peripheral nerve injury causes sensory dysfunctions that are thought to be attributable not only to the functional, cellular and biochemical events occurring in the peripheral nerve, but also to the functional changes occurring in the cerebral cortical representations of the peripheral regions....
Adaptive Control of Epileptic Seizures Using A Genetically Encoded Sensor
Principal Investigator:
Pelled, Galit
Sponsored by the National Institutes of Health (NIH) -- 1R01NS079288-01. About 35 percent of epileptic patients do not respond to antiepileptic drugs. Of these, only a quarter of them can be treated by resective surgery. Patients who have seizures arising from eloquent cortex, or which are multi-...
Accommodations for ADHD: Are They Effective in Reducing the Impact of the Disorder?
Principal Investigator:
Pritchard, Alison
One of the most important functions served by a pediatric neuropsychological assessment is the provision of evidence-based recommendations and resources designed to reduce symptoms and improve function and quality of life for youth and their families. In spite of its importance, research into the...
MRI-based Quantitative Brain Perfusion Mapping for Sickle Cell Disease
Principal Investigator:
Qin, Qin
SCD is a genetic blood disorder characterized by abnormal hemoglobin S (HbS) synthesis with sickle cell anemia (SCA) as the most common subtype. Stroke is the most frequent cause of death and long-term disability in both children and adults with SCD. Studies of patients with focal neurologic...
Pediatric Psychology and Neuropsychology Predoctoral Internship Training - Abstract
Principal Investigator:
Reeseman, Jennifer
Hearing loss or deafness affects more than 48.1 million individuals in the United States, with nearly 1 in 5 Americans displaying a hearing loss as defined by the World Health Organization criteria (Lin, Niparko & Ferrucci, 2011), and 14.9% of US children affected by hearing loss or deafness (...
ArmAware: A Novel Device to Treat Motor Disorders
Principal Investigator:
Salorio, Cynthia
The goal of this project is to develop a prototype of a novel device to treat hemiplegia. The award covers development costs of the initial prototypes.
Characterization of Biochemical Abnormalities Underlying Mitochondrial Dysfunction and Cardiomyopathy in Barth Syndrome and Adenine Nucleotide Translocase 1 (ANT-1) Deficiency
Principal Investigator:
Sandlers, Yana
The treatment of many inborn errors of metabolism is centered on recognizing and correcting, or compensating for metabolic aberrations. The limited understanding of the complexity of the biochemical aberrations in many of these disorders has hindered the ability of clinicians to compensate for the...
The IBMD-OI Study: A Study to Assess the Effectiveness of Teriparatide (FORTEO)
Principal Investigator:
Shapiro, Jay
Sponsored by Eli Lilly and Company, the primary aim of this study is to assess whether an increase in BMD will result from the use of rhPTH. Furthermore, PTH has been shown to have effects on bone structure that may be beneficial (increased cortical thickness, trabecular number, bone size) and thus...
Treatment of Osteoporosis in Murine Rett Syndrome Models
Principal Investigator:
Shapiro, Jay
Sponsored by the International Rett Syndrome Foundation. At the Kennedy Krieger Institute, approximately 50 percent of young children and adults with Rett syndrome (RTT) have osteoporosis; 11 percent of these patients have suffered fractures. RTT is associated with mutations involving MECP2 and...
Nutrition and Its Impact on Bone Growth in Children with Osteogenesis Imperfecta
Principal Investigator:
Shapiro, Jay
Sponsored by the Edward & Stella Van Houten Memorial Fund To provide information that may directly translate into improved care for children with OI and open the subject of nutrition as related to the integrity of bone in other children with poor bone development from other causes.
Kennedy Krieger Institute's Linked Clinical Research Center in Osteogenesis Imperfecta
Principal Investigator:
Shapiro, Jay
As a participant of the Linked Clinical Research Centers (LCRC), the Osteogenesis Imperfecta (or) Program at the Kennedy Krieger Institute (KKI) brings an established record of multidiscliplinary expertise in clinical care, research and education for the or community. The KKI shares firm...
Aging and Dementia in Adults with Down Syndrome
Principal Investigator:
Silverman, Wayne
This ongoing NIH supported Program Project will be renewed for another five years, and is programmatically a seamless continuation of my current Exempt protocol (#NA_00002225; "Changes in Functioning Among Mentally Retarded Adults"). The overarching objectives of this program of research will...
Pilot Treatment of the PEX1-G844D Mouse with Diosmetin
Principal Investigator:
Steinberg, Steven
We created the Pex1-G844D mouse to model the clinical phenotype found in humans associated with homozygosity for the equivalent PEX1 mutation, PEX1-G843D.  The Pex1-G844D mouse model recapitulates many features of the mild end of Zellweger Spectrum Disorders. These include elevated tissue...
Biomarkers of mild traumatic brain injury in children: predicting outcome and understanding recovery
Principal Investigator:
Suskauer, Stacy
Scientific Abstract: Current management of childhood mild TBI (mTBI, also known as concussion) is symptombased. Symptoms may last from hours to years, yet we lack objective biomarkers with which to predict outcome, inform treatment, and evaluate risk for chronic sequelae of the current or future...
Neural Basis of Recovery of Inhibitory Control After Pediatric TBI
Principal Investigator:
Suskauer, Stacy
Sponsored by NIH/NICHD - 5 K23 HD061611 Inhibitory control is frequently impaired following pediatric traumatic brain injury (TBI), and deficits in inhibitory control impact a child's function in school and at home and ultimately their likelihood for successful community integration as an adult....
Somatosensory Processing-Assessing Youth Sports-Related Concussion and Recovery
Principal Investigator:
Suskauer, Stacy
Youth sports-related concussion (SRC) is a public health problem in the United States, as evidenced by the public health approach of youth concussion laws which are now in effect in more than 40 states. Youth are felt to be particularly vulnerable to concussion, due to differences in physiological...
Novel Approaches for CEST Labeling, Detection, Quantification, and Translation
Principal Investigator:
Van Zijl, Peter
The overall Program Project focuses on the design and detection of bioorganic biodegradable Chemical Exchange Saturation Transfer (CEST) agents, with the ultimate goal of their practical application to cellular and molecular labeling and the imaging of drug delivery and gene expression in animals...
Annual Meeting of the NCRR/NIBIB Principal Investigators
Principal Investigator:
Van Zijl, Peter
We propose to continue the annual meetings of the Directors and Assistant Directors of the NCRR and NIBIB P41 Centers. The 2 1/2 day meeting is held annually in late June in Bethesda, Maryland. The meeting is also attended by individuals from the NCRR, NIBIB, and other institutes of the NIH. The...
Resource for Quantitative Functional MRI
Principal Investigator:
Van Zijl, Peter
The Resource for Quantitative Functional Magnetic Resonance Imaging is an interdepartmental and interdisciplinary laboratory combining facilities of the F.M. Kirby Research Center for Functional Brain Imaging at the Kennedy Krieger Institute (KKI) and the Center for Imaging Science (CIS) at Johns...
Biomarkers of Facioscapulohumeral Muscular Dystrophy (FSHD)
Principal Investigator:
Wagner, Kathryn R.
This NIH-funded study is established to identify biomarkers for facioscapulohumeral muscular dystrophy (FSHD). FSHD patients and their unaffected first degree relative will undergo open muscle biopsy performed at the Johns Hopkins Outpatient Surgery Center under local anesthesia. Blood samples will...
Duchenne Muscular Dystrophy: Double-Blind Randomized Trial to Find The Optimum Steroid Regimen (FOR-DMD)
Principal Investigator:
Wagner, Kathryn R.
This Phase III multi-center, double-blind, parallel group, 36 - 60 month study funded by the NIH will compare three corticosteroid regimens in wide use in DMD: 1) daily prednisone (0.75 mg/kg/day); 2) intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off); and 3) daily deflazacort (...
Myostatin regulates fate of satellite cells in dystrophic muscle
Principal Investigator:
Wagner, Kathryn R.
The goal of this proposal is to determine whether myostatin, expressed in both skeletal muscle and fibroblasts, regulates the conversion of satellite cells to fibroblasts and whether this conversion contributes to fibrosis prominent in dystrophic muscle. The knowledge obtained by this study will...
A Three Dimensional Environment for Skeletal Muscle Stem Cell Transplantation
Principal Investigator:
Wagner, Kathryn R.
The purpose of this collaborative project is to develop and test and injectable scaffold embedded with muscle stem cells and a Pfizer drug to facilitate muscle regeneration. Cell based therapies lag behind the development of other approaches to chronic muscle disease including gene therapy and...
Biomarkers for Therapy of FSHD
Principal Investigator:
Wagner, Kathryn R.
The goal of this Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center is to define modifying genes of FSHD and to determine, through novel animal models of FSHD, whether these are appropriate therapeutic targets. 1) Our current research will seek to identify genetic modifiers...
Magnetic Resonance Spectroscopy Imaging as a Biomarker for FSHD
Principal Investigator:
Wagner, Kathryn R.
Sponsored by the American Academy of Neurology -- 2011 AAN Clinical Research Training Fellowship. Recent advances in the study of heritable neuromuscular disorders have led to multiple promising therapeutic approaches to formerly untreatable, and sometimes fatal, disorders. As clinical trials...
Acupressure and Acupuncture as an Intervention with Children with Autism II
Principal Investigator:
Warren, Lana
The purpose of this feasibility study is to see if children with autism spectrum disorder (ASD) will tolerate an acupressure and acupuncture intervention twice weekly over a 3 month period of time that targets regulatory and behavioral functions. It is anticipated that: 1) children with ASD will...
Acyl-CoA Synthetase ACSVL3 in Malignant Glioma: Metabolism and Oncogenic Cellular
Principal Investigator:
Watkins, Paul
Malignant glioma accounts for a significant percentage of brain tumors. As these tumors are typically refractory to treatment, there is a need for new and novel therapeutic approaches. Levels of ACSVL3, an enzyme of fatty acid (FA) metabolism, were found to be highly upregulated in human malignant...
Modeling Aggressive Medulloblastoma Using Human Induced Pluripotent Stem Cells
Principal Investigator:
Ying, Mingyao
Brain tumors are the most common cause of childhood oncological death, and medulloblastoma (MB) is the most common malignant pediatric brain tumor. Group 3 MB is characterized by amplification and overexpression of oncogenic transcription factor MYC, herein referred to as MYC-driven MB. Modeling...
Understanding Mechanisms of Lower Extremity Strengthening in Women Heterozygous for X-ALD
Principal Investigator:
Zackowski, Kathleen
Sponsored by the ELA Foundation. X-linked adrenoleukodystrophy (X-ALD), a sex-linked progressive neurodegenerative disease, is caused by a defect in the ABCD1 gene. The disease is expressed in multiple ways, but the most common adult form is adrenomyeloneuropathy (AMN), which results in slowly...
Predicting Intervention Responsiveness to Improve Rehabilitation in People with Multiple Sclerosis
Principal Investigator:
Zackowski, Kathleen
Sponsored by the National Multiple Sclerosis Society -- RG 4668-A-1. Multiple sclerosis (MS) is an inflammatory disease of the central nervous system (CNS) distinguished pathologically by inflammatory demyelination and axonal injury. It is characterized by a progressive functional decline although...
Mechanisms of Locomotor Recovery in Multiple Sclerosis
Principal Investigator:
Zackowski, Kathleen
The study will test the general hypothesis that, in multiple sclerosis (MS) and following an acute exacerbation, damage of cerebellar white matter tracts (i.e. cerebellar peduncles) will show greater gait ataxia and worse recovery of walking compared to people with damage to motor cortex tracts (i....
Sleep Phenotypes in Children
Principal Investigator:
Accardo, Jennifer
Children have a high prevalence of sleep problems and disorders. The current study will be a retrospective record review of clinic records from a subspecialty sleep medicine clinic staffed by a child neurologist who is board certified in Sleep Medicine and a behavioral psychologist. No other data...
Use of an Educational Story to Prepare Children with Developmental Disabilities for Overnight Polysomnography
Principal Investigator:
Accardo, Jennifer
We hypothesize that use of an educational story with pictures illustrating overnight polysomnography procedure accompanied by simple narrative will be a cost-effective, readily accepted intervention that will contribute to successful completion of polysomongraphic procedures among children with...
Addressing Sleep Problems in Children with Autism Spectrum Disorder at Multidisciplinary Autism Centers
Principal Investigator:
Accardo, Jennifer
Sleep problems are highly prevalent among children with autism spectrum disorders (ASD), particularly insomnia. Pediatric sleep problems are often overlooked in typically-developing children. It is unknown how often they are addressed in children with ASD. This project is a retrospective review of...
Neuropsychological Functioning in Demyelinating Diseases
Principal Investigator:
Arenivas, Ana
Demyelinating diseases are rare neurological autoimmune conditions characterized by inflammatory processes in which the myelin sheath surrounding axons within the central nervous system (CNS) is damaged. In the pediatric population, these conditions may be monophasic or chronic. Typical monophasic...
Neuroimaging and Neurobehavioral Outcomes of Pediatric Neuromyelitis Optica: A Pilot Study
Principal Investigator:
Arenivas, Ana
The primary objective of this proposed prospective pilot study, entitled “Neuroimaging and Neurobehavioral Outcomes of Pediatric Neuromyelitis Optica: A Pilot Study” is to determine the utility of specific neuroimaging modalities as biomarkers of Neuromyelitis Optica (NMO) using MPRAGE, diffusion...
The Role of Cerebellar Hyperactivity in Parkinson's Disease
Principal Investigator:
Bastian, Amy
The purpose of this work is to study the role of the cerebellum in gait dysfunction for Parkinson's disease (PD) patients. While the symptoms of PD were originally ascribed to dopamine deficiency and basal ganglia dysfunction, recent research has shown that the cerebellum is hyperactive in PD...
Long Term Split Belt Training for Stroke Recovery
Principal Investigator:
Bastian, Amy
We recently discovered that the walking deficits of people with post-stroke hemiparesis can be improved on short timescales by adaptation to split-belt treadmill conditions that exaggerate the deficit. The exaggeration drives the nervous system to adapt to correct the deficit, and results in...
Locomotor Control and Learning
Principal Investigator:
Bastian, Amy
In this project, we will study split belt treadmill walking and walking under natural conditions. We have found that adaptation to split belt walking in adults and children varies in its generalization to other forms of locomotion, as well as other contexts and other speeds. Our previous research...
Cerebellar Control of Movement
Principal Investigator:
Bastian, Amy
Cerebellar damage disrupts movement coordination. Actions ranging from walking to reaching become inaccurate and difficult to control, which has deleterious effects on most functional movements. Therapeutic approaches for cerebellar motor disorders (i.e. 'ataxia') rely heavily on rehabilitation...
Locomotor Training for Neurological Disease
Principal Investigator:
Bastian, Amy
For this pilot study our aims are to:  Understand basic brain mechanisms of inter-limb control and adaptation during locomotion to determine the paradigm to be used for improving interlimb coordination Determine how the pattern (e.g. which leg is made to move fast) influences...
Exercise Facilitates Learning
Principal Investigator:
Bastian, Amy
The purpose of this work is to study the effect of a single, acute bout of physical exercise on subsequent motor learning. Previous studies have shown that acute bouts of aerobic exercise can lead to improvement on cognitive learning and memory tests. We hypothesize that aerobic exercise will also...
The Utility of Interdisciplinary Model in Evaluating Developmental Delays in Preschool Developmental Clinic
Principal Investigator:
Bay, Mihee
Compare interdisciplinary and single consult models of preschool developmental evaluations for: Length of time from first to last consultation Number of diagnoses Number of referrals in children with Autism for ADOS
FIRST-STIM CNS Growth Factor Release and Changes in the Inflammatory Environment in Response to Electrical Stimulation in Subjects with Inflammatory Myelopathies
Principal Investigator:
Becker, Daniel
A Phase II Randomized, Single-Blinded, 100-Subject Clinical Trial of Functional Electrical Stimulation in the Treatment of Inflammatory Myelopathies The specific aims of the proposal are: To determine which functional elecrical stimulation (FES) ergometry dosing regimen results in the most...
The Development of a Reading Intervention for Deaf and Hard-of-Hearing Students
Principal Investigator:
Beetar, John
The specific aim of this project is to complete the development of a computer software program of reading instruction, Sign 'n' Read, for deaf and hard-of-hearing students. Presently in development as a prototype, Sign 'n' Read will consist of two modules, a sight-word module and a vocabulary...
KKI TFC KIDnet Outcome Monitoring System
Principal Investigator:
Belcher, Harolyn
The KIDnet system is a clinical needs assessment, functional outcome measure, and therapeutic effectiveness database used by the State of Maryland and clinical foster care providers (Kennedy Krieger Institute Therapeutic Foster Care -- KKI TFC) under the auspices of University of Maryland,...
Strengthening Families' Coping Resources Open Trial
Principal Investigator:
Belcher, Harolyn
This study is an open trial to determine the effectiveness of the Strengthening Family Coping Resources (SFCR) clinical treatment for children and families affected by trauma exposure. The clinicians at the Center for Child and Family Traumatic Stress at Kennedy Krieger Institute have participated...
Kennedy Krieger Institute Center for Child and Family Traumatic Stress - Treatment Effectiveness Study (KKFC-TES)
Principal Investigator:
Belcher, Harolyn
The goal of this study is to determine the effectiveness of the various programs within the Kennedy Krieger Center for Child and Family Traumatic Stress using data from a computerized clinical database. Established in 1985, the KKFC is a high volume community-based mental health clinic that...
RISE: Promoting Diversity among Public Health Professionals
Principal Investigator:
Belcher, Harolyn
This IRB proposal is to obtain permission to publish the outcome results of a student training program to promote public health careers among under-represented graduate students. All data have been previously collected and are stored within the training program database. We are requesting...
MCHC/RISE-UP and Ferguson Fellowship Program Evaluation
Principal Investigator:
Belcher, Harolyn
The proposed study is an federally mandated evaluation of two CDC-funded public health training programs. The overarching goal is of the programs is to increase the diversity of the public health work force. Primary outcomes are student public health exposure, knowledge, and career choice.
Accessing Outcomes of SMART:Safety Mentoring Advocacy Recovery and Treatment Model
Principal Investigator:
Belcher, Harolyn
This is a study of children evaluated and treated using the SMART Model at the Center for Child and Family Traumatic Stress at Kennedy Krieger Institute. The Center for Child and Family Traumatic Stress provides trauma and evidence-informed mental health services for children exposed to adverse...
Understanding the Associations between Trauma Exposure and Child Behaviors
Principal Investigator:
Belcher, Harolyn
This is a study of children evaluated and treated at the Center for Child and Family Traumatic Stress at Kennedy Krieger Institute. The Center for Child and Family Traumatic Stress provides trauma and evidence-informed mental health services for children exposed to adverse childhood experiences (...
Urinary Tract Infection in Kennedy Krieger Institute Patients
Principal Investigator:
Belcher, Harolyn
Urinary tract infections (UTI) are one of the most common bacterial infections (Schappert & Rechtsteiner, 2008; Litwin et al., 2005). Data from the National Ambulatory Medical Care Survey estimated that UTI caused 8.1 million physicians visits (Schappert & Rechtsteiner, 2008). Children...
National Child Traumatic Stress Network (NCTSN) Quality Improvement Initiative Database
Principal Investigator:
Belcher, Harolyn
The proposed study is to request permission to continue federally mandated data collection to examine the clinical outcomes of children at the Center for Child and Family Traumatic Stress at Kennedy Krieger Institute who are enrolled in evidence-based and promising trauma-informed treatments as...
The Garden of Hope: Horticultural Therapy with Traumatized Children
Principal Investigator:
Belcher, Harolyn
The Garden of Hope: Horticultural Therapy with Traumatized Children (HTTC) study will evaluate the effectiveness of horticultural therapy as an adjunct therapy to standard mental health treatment for traumatized children, ages 6-17 years, at the Kennedy Krieger Center for Child and Family Traumatic...
TF-CBT Effectiveness Study
Principal Investigator:
Belcher, Harolyn
Trauma Focused Cognitive Behavior Therapy (TF-CBT) is an evidence-based mental health treatment designed for children who have experienced traumatic life events. Children at the Kennedy Krieger Center for Child and Family Traumatic Stress receive TF-CBT on a clinician available basis. The TF-CBT...
KKI Benchmark TFC
Principal Investigator:
Belcher, Harolyn
The Kennedy Krieger Institute (KKI) Benchmark Treatment Foster Care (TFC) evaluation study will collect program evaluation and patient demographic, treatment and eight outcome measures on the KKI TFC programs. This is longitudinal study to evaluate treatment outcomes of children enrolled in TFC in...
NCTSI Cross-Site Evaluation Component 1 and 2
Principal Investigator:
Belcher, Harolyn
What are the descriptive and clinical outcomes of 25 children treated at KKFC? Child-Caregiver dyads treated at KKFC will be assessed at baseline, three-months, six-months, nine-months and 12-months. What opinons and satisfaction level do caregivers have concerning pediatric treatment services...
Yoga Based Movement Therapy Study
Principal Investigator:
Belcher, Harolyn
This preliminary study will examine the feasibility of conducting a randomized clinical trial of Yoga Based Movement Therapy (YBMT)and standard care mental health (SCMH) treatment compared to SCMH for children ages 8-12 years who have a history of neglect and maltreatment (trauma). The goal of the...
KKFC Children at Risk Study
Principal Investigator:
Belcher, Harolyn
This study is designed to evaluate the association between parental risk factors and child behavior and parental stress following six months of mental health treatment. The study will use data from a previously IRB approved electronic database protocol. Data analysis will be performed on a limited...
Child Attitudes Towards Drug Use Risk
Principal Investigator:
Belcher, Harolyn
Examine the responses of children treated at the JHH Adolescent clinic towards drug use and drug use risk. Baseline responses of children, ages 10-14 years, on a Project Alert: Survey of Student Attitudes and Behaviors will be compared between a group of children whose mothers are in a drug...
The Baltimore Behavioral Enhancement through Training & Teaching to Expand Resiliency (BETTER) Family & Community Partnership
Principal Investigator:
Belcher, Harolyn
Does Head Start Center-based mental health and substance abuse prevention intervention improve parent, child, and family outcomes (e.g. child behavior, parent stress and mental health, family cohesion, utilization of community resources, etc.)? Do parents in the study with a history of...
FamilyLive Feasibility and Effectiveness Study
Principal Investigator:
Belcher, Harolyn
Children who have been exposed to neglect often have parents who experienced their own childhood neglect and/or trauma (defined as childhood physical, sexual or emotional abuse, exposure to life threatening events, domestic and/or community violence, parental mental health disorders, substance...
Leadership Behaviors of Health Professionals and the Associations with Knowledge, Attitudes, and Practices Related to Health Promotion for Individuals with Developmental Disabilities
Principal Investigator:
Belcher, Harolyn
This study, using quantitative survey methodology, will assess health professionals working in University Centers for Excellence in Developmental Disabilities (UCEDD) to measure leadership behaviors and health promotion practices. The outcome variables are knowledge, attitudes, and practices of...
Relationship of MRI to ASIA Impairment Scale in Chronic Spinal Cord Injury
Principal Investigator:
Belegu, Visar
Structural & functional MRI studies of the central & peripheral nervous system in healthy children & children with spinal cord injury at 1.5 & 3.0 T Development of imaging correlates of the integrity of spinal cord white matter is needed for optimizing design of clinical trials...
A Comparison of Two Physical Guidance Procedures in the Treatment of Pediatric Food Refusal
Principal Investigator:
Borrero, Carrie
Escape prevention procedures implemented in the treatment of food refusal may vary across clinical settings. An evidence base exists for at least one form of physical guidance that is commonly implemented in the treatment of food refusal. The jaw prompt (i.e., application of slight pressure to the...
Conceptualizing Food Preferences as Rich-to-Lean and Lean-to-Rich Transitions: A Translational Analysis
Principal Investigator:
Borrero, Carrie
The purpose of this study is to determine if a children's food preferences following treatment for pediatric food refusal continue to impact mealtimes. During meals, typically multiple foods are presented, and this often includes both preferred and nonpreferred foods. In some cases, in clinical...
Some Effects of Caregiver Integrity Given the Initiation of Training
Principal Investigator:
Borrero, Carrie
Behavioral treatment of pediatric feeding disorders focuses on weakening or eliminating environmental contingencies that support food refusal. Previous descriptive research evaluating parent-child interactions related to food refusal has identified removal of the spoon/cup, parent attention, and...
Examining Child Mealtime Behavior Following the Implementation of Behavioral Treatments for Food Refusal
Principal Investigator:
Borrero, Carrie
Behavioral treatment of pediatric feeding disorders focuses on weakening or eliminating environmental contingencies that support food refusal and arranging contingencies to increase the likelihood of appropriate mealtime behavior (e.g., accepting bites, swallowing). Previous research has found...
An Evaluation of Noncontingent Reinforcement and Differential Reinforcement with Escape Extinction in the Treatment of Food Refusal
Principal Investigator:
Borrero, Carrie
This study is a retrospective record review of former patients on the Pediatric Feeding Disorder Unit at Kennedy Krieger Institute. The primary objective of this study is to compare effects of escape extinction with and without positive reinforcement to decrease inappropriate mealtime behavior and...
Descriptive Analyses of emesis and an experimental evaluation of the effects of NCR on emesis
Principal Investigator:
Borrero, Carrie
Pediatric feeding disorders can result in severe consequences such as growth failure. Feeding disorders are also associated with a wide range of different types of refusal. Food refusal can include vomiting; however, interventions to target vomiting are underreported in the current feeding...
The Use of Naturally Occurring Schedules of Reinforcement to Decrease Inappropriate Mealtime Behavior: Are General Recommendations Enough?
Principal Investigator:
Borrero, Carrie
The primary objective of this research is to determine if an approximation of natural schedules of reinforcement (observed during caregiver-fed meals with children) can be approximated through the use of conditional probability and lag sequential analyses. All data collected will be retrospective...
The Use of Video Modeling via an iPad to Increase Independence with Vocational Tasks for Students with Autism
Principal Investigator:
Brandenburg, Linda
The purpose of this study is to employ a single-case reversal research design (ABAB) to evaluate the effectiveness of using video modeling procedures by means of an iPad to increase independent task completion for five to six students with autism spectrum disorders enrolled in a special education...
Fragile X Clinical and Research Cooperative Consortium Registry and Repository
Principal Investigator:
Budimirovic, Dejan
This project will advance the core activities of the Fragile X Clinical and Research Consortium (FXCRC). The FXCRC is a collaborative endeavor initiated in 2006 by the National Fragile X Foundation (NFXF) to advance clinical practice and facilitate coordinated, collaborative multi-site research on...
Early signs of Fragile-X syndrome
Principal Investigator:
Budimirovic, Dejan
The proposed study intends to begin identifying early signs of deviation from typical development (i.e., motor, language/communication, social) in individuals with FXS. Our study will be based on the retrospective analysis of family audio-video recordings of individuals with FXS. The study design...
Fragile-X Clinic Pharmacological Database
Principal Investigator:
Budimirovic, Dejan
This project will collect retrospective data in order to establish a research database containing pharmacological parameters from medical records of patients of the Fragile X Clinic at Kennedy Krieger Institute. The database is to be used for management and storage of pharmacological data for the...
High Resolution FMR1 Genetic and Epigenetic Molecular Assessments in a Well Characterized Cohort of Full Mutation and Premutation Fragile X Patients
Principal Investigator:
Budimirovic, Dejan
The fragile X gene, FMR1, is critical for brain development and has many markers. Syndromic autism spectrum disorder (ASD) is common in males with an FMR1 full mutation (FM, >200 CGG). Yet males with the premutation (PM, 55-200 CGG) may also represent a significant form of FMR1 relevant to ASD...
Survey of Academic Performance in Children with a History of Neonatal Encephalopathy
Principal Investigator:
Burton, Joanna
The proposed study is a telephone survey for parents of children who were treated with therapeutic hypothermia in the Johns Hopkins NICU. It is designed to provide descriptive data regarding early school age outcomes after perinatal hypoxic ischemic injury treated with hypothermia and allow...
Depression and Sleep Disorders in Adolescents and Young Adults with Down Syndrome
Principal Investigator:
Capone, George
Persons with Down syndrome (DS) are known to exhibit adolescent and adult-onset psychopathology such as depression, anxiety, obsessive-compulsive and schizophreniform disorders. Many also have medical conditions which increase their risk for developing sleep-related breathing disorders (SRBD)....
Neuropsychiatric disorder in young adults with Down syndrome
Principal Investigator:
Capone, George
We propose to consecutively enroll new patients presenting to our clinic for initial clinical evaluation of neuropsychiatric and behavioral problems. Our goal is to 1) quantify psychiatric and behavioral symptomatology using standardized measures, 2) obtain a detailed family mental health history...
A 20-Week Double Blind Placebo Controlled Clinical Trial to Evaluate the Safety and Efficacy of Rivastigmine in Children (ages 10-17) with Down syndrome
Principal Investigator:
Capone, George
The specific aims of this study are to: a) investigate short-term safety and efficacy of rivastigmine treatment; b) build upon the open-label treatment results of overall function and language improvement in children with Down syndrome (DS) in a double-blind, placebo-controlled clinical trial; and...
Therapeutic Hypothermia After Pediatric Cardiac Arrest: The THAPCA Trials (Telephone Follow Up Center)
Principal Investigator:
Christensen, James
Cardiac arrest in childhood often results in death or poor quality neurologic survival. Out-of-hospital cardiac arrest in children is usually preceded by respiratory failure and hypoxia, whereas in-hospital cardiac arrest is usually preceded by hypotension. Survival rate and neurologic outcomes are...
Developing a Means of Comparing Expenditures at Three Models of Nonpublic Special Education Services at a Private Special Education School in Baltimore, Maryland
Principal Investigator:
Church, Robin
Currently, there is no uniform means of determining the true cost of nonpublic special education in Maryland. Today, tuition is used as “the cost” of providing these programs. The proposed project will identify a formula that nonpublic schools and Local Education Agencies may use to determine per...
Innovative approaches to gauge neurologic progression in Sturge-Weber syndrome
Principal Investigator:
Comi, Anne
This 1 of three projects funded by a NIH consortium grant to study Sturge-Weber syndrome (SWS). It will create a national database, determine if urine biomarkers can predict neurologic progression in SWS, and apply DNA arrays to the problem of discovering the putative somatic mutation hypothesized...
Multidiciplinary Protocol to Address the Pathophysiology of Sturge-Weber Syndrome
Principal Investigator:
Comi, Anne
The Multidisciplinary Protocol to Address the Pathophysiology of Sturge-Weber Syndrome's goal is to better understand how Sturge-Weber Syndrome affects the brain, eyes, skin, blood vessels, behavior, and psychology. We hope to gather information that can lead to a discovery of the cause of Sturge-...
Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber syndrome
Principal Investigator:
Comi, Anne
The primary objective of this study is to determine the tolerability and optimal dose of cannabidiol (CBD) as an adjunct treatment in children and young adults with Sturge-Weber syndrome and drug resistant epilepsy. To begin to assess this we will do a small expanded access study with 5 subjects...
Qualitative interviews and focus groups with military-connected parents
Principal Investigator:
Crockett, Jennifer
What do military families identify as key considerations and barriers in seeking treatment for their children with emotional and behavioral challenges? Our plan is to conduct individual interviews and focus groups with parents of military connected children who sought behavioral services.
Studies of the Link between Autism and Epilepsy
Principal Investigator:
Ewen, Joshua
This research consists of a retrospective record review to examine the link between autism, epilepsy and other clinical symptoms (including, but not limited to, intellectual disability, motor function and the presence/severity of psychological disorders). Our primary intent is to generate...
Electrophysiological Studies of Attention and Perception
Principal Investigator:
Ewen, Joshua
This protocol is designed to allow for the recruitment of healthy adult volunteers in order to perform studies of attention and perception in human cognition. The primary purpose of this data collection is to advance the current knowledge of human cognition and the relationship between the brain...
Pilot Studies for Event-Related Potential Experiments
Principal Investigator:
Ewen, Joshua
This protocol is designed to allow for the recruitment of healthy adult volunteers in order to pilot new paradigms for event-related potential (ERP) research. Since a significant portion of the research conducted in the Kennedy Krieger Institute Clinical Neurophysiology Laboratory uses volunteers...
Electrophysiological Studies of Autism
Principal Investigator:
Ewen, Joshua
Abnormalities of cortical connectivity have long been suspected to be responsible, at least in part, for the autism phenotype. Research to date has used a number of modalities, but none has the time resolution and the ability to detect physiologically important oscillations of EEG. We plan to...
Patient attitudes toward informed consent regarding incidental findings in whole exome sequencing
Principal Investigator:
Fatemi, S. Ali
The objective of this study is to assess the association between school-based transition planning and health care transition planning for youth with special healthcare needs (YSHCN). The 2011 Maryland Youth Transition Survey, conducted in 2012, will be used to perform a cross-sectional analysis...
Genetic Causes of Intellectual Disability
Principal Investigator:
Fatemi, S. Ali
The purpose of this study is to define the biochemical and genetic basis of peroxisomal disorders and to develop new methods for prevention and treatment. The study obtains samples of blood or skin fibroblasts from subjects with peroxisomal disorders for genetic and biochemical analysis.
ALD Connect
Principal Investigator:
Fatemi, S. Ali
Adrenoleukodystrophy (ALD) is an X-linked disorder with several phenotypes ranging from childhood to adulthood. The objective of ALD Connect is to create a patient-centered clinical research network for ALD allowing researchers to conduct patient-centered comparative effectiveness research studies...
Masqueraders of Cerebral Palsy
Principal Investigator:
Fatemi, S. Ali
Genetic conditions that manifest movement and postural impairment at perinatal or early childhood periods are often diagnosed with cerebral palsy. Identifying masqueraders of cerebral palsy represents an advance in medicine, as a number of these genetic disorders are treatable and significantly...
A Multi-Center, Randomized, Double-Blind, 12-Week, Parallel Group, Placebo-Controlled Proof of Concept Study to Investigate the Efficacy and Safety of RO5285119 in Individuals with Autism Spectrum Disorders (ASD)
Principal Investigator:
Findling, Robert
The main objective of this study is to evaluate the efficacy of 12-week treatment with RO5258119 compared with placebo in treating social communication deficits in adult individuals with ASD as measured by the social responsiveness scale (SRS) as well as to evaluate the safety and tolerability...
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Fixed Dose Study of Nnz-2566 in Fragile X Syndrome
Principal Investigator:
Findling, Robert
The purpose of this study is to look at the safety of NNZ-2566 for adolescents and adults with Fragile X Syndrome and to determine the drug properties (pharmacokinetic) of NNZ-2566 in people with Fragile X syndrome. The study will also investigate measures of efficacy during treatment.
VLZ-MD-21: A Double-Blind, Placebo-Controlled Evaluation of the Safety and Efficacy of Vilazodone in Adolescent Patients with Major Depressive Disorder
Principal Investigator:
Findling, Robert
Purpose This is a double-blind, randomized, placebo-controlled, fixed-dose trial designed to evaluate the efficacy, safety, and tolerability of vilazodone for the treatment of major depressive disorder in adolescents (ages 12-17, inclusive). A secondary goal is to utilize pharmacokinetic data...
A Randomized, Placebo-Controlled Study to Investigate the Efficacy and Safety of Circadin® to Alleviate Sleep Disturbances in Children with Neurodevelopmental Disabilities
Principal Investigator:
Findling, Robert
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy of a double-blind, 13 week treatment period with Circadin 2/5mg in...
A Randomized, 6-Week, Double-Blind, Placebo-Controlled, Flexible Dose, Parallel-Group Study To Evaluate The Efficacy And Safety Of Lurasidone In Children And Adolescent Subjects With Bipolar I Depression
Principal Investigator:
Findling, Robert
To evaluate the efficacy of lurasidone (20 - 80 mg/day flexibly dosed) compared with placebo in children and adolescent subjects with bipolar I disorder, most recent episode depressed, with or without rapid cycling disease course, and without psychotic features (diagnosed by Diagnostic and...
A Phase Ill, Randomized, Double-Blind, Placebo­ Controlled, Parallel-Arm, Multi-Center Study Measuring the Efficacy and Safety of Prc-063 in Adolescent ADHD Patients
Principal Investigator:
Findling, Robert
This is a parallel, randomized, double-blind, forced-dose titration, multicenter, placebo­ controlled, phase 3 study to evaluate the safety and efficacy of PRC-063 (methylphenidate hydrochloride controlled-release capsules 25, 45, 70 or 85 mg/day) in the treatment of Attention­ Deficit/...
A 6-Week, Randomized, Multicenter, Double-Blind, Parallel, Flexed- and Fixed-Dose Study Of Mg01ci (Metadoxine Extended-Release) 700 Mg and 1400 Mg Compared With Placebo in Adults and Adolescents With Fragile X Syndrome
Principal Investigator:
Findling, Robert
The study medication, MG01CI (Metadoxine Immediate-release/Slow-release Bilayer Tablet), is being investigated for the treatment of Fragile X Syndrome (FXS) in adults and adolescents. The purpose of this clinical study is to study how well MG01CI (Metadoxine immediate-release/slow-release, bilayer...
A Phase 1, Multicenter, Open-label, Dose-Escalation Trial to Assess the Safety, Tolerability and Pharmacokinetics of Oral Brexpiprazole (OPC-34712) in Adolescents with Schizophrenia
Principal Investigator:
Findling, Robert
This is a phase 1, multicenter, open-label, dose-escalation trial in adolescent subjects (age 13 to 17 years) with a diagnosis of schizophrenia. This trial consists of a screening period (3 to 42 days, including washout of prohibited medications), a Dose Titration Phase, Fixed Dose Phase, and a...
A Phase 3, 12-Month, Multicenter, Open-label, Flexibly-dosed, Safety Study of SEP-225289 in Adults with Attention Deficit Hyperactivity Disorder (ADHD)
Principal Investigator:
Findling, Robert
To evaluate the long-term safety and tolerability of SEP-225289 in adult subjects with ADHD by the incidence of adverse events (AEs; or serious AEs), AEs (or SAEs) leading to discontinuation.
A 6-Month Study to Evaluate the Safety, Tolerability and Efficacy of Desvenlafaxine Succinate Sustained-Release (Dvs Sr) Tablets in the Treatment of Children and Adolescent Outpatients with Major Depressive Disorder
Principal Investigator:
Findling, Robert
A 6-Month, Open-Label, Multi-Center, Flexible-Dose Extension Study to the B2061032 Study to Evaluate the Safety, Tolerability and Efficacy of Desvenlafaxine Succinate Sustained-Release (Dvs Sr) Tablets in the Treatment of Children and Adolescent Outpatients with Major Depressive Disorder To...
A Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety and Tolerability of Desvenlafaxine Succinate Sustained-Release (Dvs Sr) in the Treatment of Children and Adolescent Outpatients with Major Depressive Disorder
Principal Investigator:
Findling, Robert
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety and Tolerability of Desvenlafaxine Succinate Sustained-Release (Dvs Sr) in the Treatment of Children and Adolescent Outpatients with Major Depressive Disorder To evaluate the...
A Randomized, Double-Blind, Multicenter, Placebo-controlled, Parallel-group, Efficacy and Safety Study of 2 Doses of Dasotraline in Adults with Attention Deficit Hyperactivity Disorder (ADHD)
Principal Investigator:
Findling, Robert
To evaluate the efficacy and safety of dasotraline (4mg/day and 6 mg/day) compared with placebo in adult subjects with ADHD as measured by change from baseline at Week 8 in ADHD symptoms assessed using the ADHD Rating Scale Version IV (ADHD RS-IV) with adult prompts total score.
D1050302: A 104-week, flexible-dose, open-label, multicenter, extension study to evaluate the long-term safety and effectiveness of Lurasidone in pediatric subjects with schizophrenia and subjects with irritability associated with autistic disorder
Principal Investigator:
Findling, Robert
This open-label extension study is designed to evaluate the safety, tolerability, and effectiveness of lurasidone for the treatment irritability in individuals with autistic disorder. As an extension of NA_00089697, this 104-week study will provide an adequate time frame to evaluate the long-term...
A Multicenter, Double-blind, Placebo- and Active-Controlled Parallel-Group Evaluation of the Safety and Efficacy of Vilazodone in Pediatric Patients With Major Depressive Disorder
Principal Investigator:
Findling, Robert
The objective of this study is to evaluate the efficacy, safety, and tolerability of vilazodone compared with placebo in pediatric outpatients (7-17 years of age) with MDD. In addition, the study is designed to obtain pharmacokinetic (PK) data to define the PK profile of vilazodone in the...
A Phase 3, multicenter, three-week, randomized, double-blind, placebo-controlled, parallel-group efficacy and safety trial of flexible doses of oral Ziprasidone in children and adolescents with Bipolar I disorder (current or most recent episode manic)
Principal Investigator:
Findling, Robert
This will be a Phase 3, multicenter, 3-week, randomized, double-blind, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and tolerability of flexibly dosed ziprasidone compared with placebo for the treatment of Bipolar I Disorder. 
12708A: An open-label study evaluating the Pharmacokinetics and Tolerability of Lu AA21004 in connection with multiple oral dosing of Lu AA21004 in child and adolescent patients with a DSM-IV-TR(TM) diagnosis of Depressive or Anxiety Disorder
Principal Investigator:
Findling, Robert
This open-label study will evaluate the pharmacokinetic, tolerability and safety profiles of Lu AA21004 in connection with a multiple oral dosing in child and adolescent patients with a DSM-IV-TR diagnosis of Depressive or Anxiety Disorder. This study also aims to support further development of...
Interventional, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of Vortioxetine in Adults with Attention Deficit Hyperactivity Disorder (ADHD)
Principal Investigator:
Findling, Robert
Primary Objective: To evaluate the efficacy of vortioxetine 10 mg/day and 20 mg/day versus placebo on ADHD symptoms in the treatment of adult patients with a DSM-5™ diagnosis of ADHD. Secondary Objectives: To evaluate the efficacy of vortioxetine 10 mg/day and 20 mg/day versus...
An adaptive, phase IIB/III, double-blind, randomized, placebo-controlled, multi-center study of the safety and efficacy of NaBen® (sodium benzoate), a d-amino acid oxidase inhibitor, as an add-on treatment for schizophrenia in adolescents
Principal Investigator:
Findling, Robert
Phase IIb Objectives: The primary objective of the Phase IIb portion of this study is to evaluate NaBen® (sodium benzoate) (1000 mg/day) safety and effectiveness and to determine the final sample size needed in order to proceed with the Phase III part of this trial. The secondary objective of this...
A0501093: SPRITES: SERTRALINE PEDIATRIC REGISTRY FOR THE EVALUATION OF SAFETY: A Non-Interventional, Longitudinal, Cohort Study to Evaluate the Effects of Long-Term Sertraline Treatment in Children and Adolescents
Principal Investigator:
Findling, Robert
The primary objectives are to evaluate the long-term impact of treatment with sertraline on aspects of cognition, emotional and physical development, and pubertal maturation. Secondary objectives include: (1) evaluating the relative risks and benefits of long-term sertraline exposure and (2)...
A Phase 2, Exploratory Safety, Tolerability, Pharmacokinetic, and Efficacy Study of SPD489 in Preschool Children Aged 4-5 Years with Attention-Deficit/Hyperactivity Disorder (ADHD)
Principal Investigator:
Findling, Robert
A Phase 2, Open-label, Multicenter, Exploratory Safety, Tolerability, Pharmacokinetic, and Efficacy Study of SPD489 in Preschool Children Aged 4-5 Years with Attention-Deficit/Hyperactivity Disorder (ADHD) To evaluate the safety and tolerability of SPD489, administered as a daily morning dose (...
Validation Study of the Aggression Diary
Principal Investigator:
Findling, Robert
The primary objective of this study is to conduct psychometric tests on the electronic observer- reported (eObsRO) Aggression Diary to evaluate whether the diary is a valid and reliable measure for assessing the benefit of SPN-810 (Molindone hydrochloride extended-release tablets) for the treatment...
A Multi-Country Analysis of Patients with Chronic Myeloid Leukemia
Principal Investigator:
Gary, Devin
This application is based on a study that was originally carried out and completed at George Washington University. All of the data for this study has been collected. No new data will be collected at JHU or KKI. In addition, all of the data that has been, or will be analyzed is /will be completely...
Retrospective Chart Review of Growth in Osteogenesis Imperfecta
Principal Investigator:
Germain-Lee, Emily
The purpose of this research is to assess the growth parameters(height and weight) in osteogenesis imperfecta during infancy, childhood, and adolescence with the ultimate goal of determining whether there are key points in time during which growth is impaired. We will also tabulate adult heights...
Studies of Hormone Action in Patients with Altered G-protein-coupled Signal Transduction
Principal Investigator:
Germain-Lee, Emily
Includes Substudies Entitled: "Study of Growth Hormone Secretion in Patients with Pseudohypoparathyroidism Type 1a (R01 FD002568)" and "Phase 2 Growth Hormone for Treatment of Albright Hereditary Osteodystrophy (R01 FD003409)" Albright hereditary osteodystrophy (AHO) is a rare genetic disorder...
Predictive Value of Perinatal Biomarkers Following Very Preterm Birth for Cerebral Palsy and Developmental Delay at Age 24 Months
Principal Investigator:
Gerner, Gwendolyn
Neurologic injury identified in neonates soon after pre-term birth may either completely resolve or lead to life-long disabilities, including cerebral palsy (CP), that are a severe burden for these patients and their families. There is currently much focus on very early identification of neurologic...
Interdisciplinary Protocol for Patients with TSC
Principal Investigator:
Gipson, Tanjala
The major features of Tuberous Sclerosis Complex (TSC) are abnormalities of the brain –cortical tubers, subependymal nodules and subependymal giant cell astrocytomas; skin –hypomelanotic macules, shagreen patches, facial angiofibromas, and ungual fibromas; kidneys – renal hamartomas or...
Rapalogues for Autism Phenotype in TSC: A Feasibility Study
Principal Investigator:
Gipson, Tanjala
Tuberous sclerosis complex (TSC) is associated with autism and seizures. Recent studies in animals using rapamycin have shown cognitive improvement. Human studies have demonstrated a reduction in tumor burden, subjective cognitive improvement, and decreased seizure frequency. The goal of this study...
Rapamycin for Autism Phenotype & seizures in Tuberous Sclerosis (RAPS)
Principal Investigator:
Gipson, Tanjala
Tuberous sclerosis complex (TSC) is associated with autism and seizures. Recent studies in animals using rapamycin have shown cognitive improvement. Human studies have demonstrated a reduction in tumor burden, subjective cognitive improvement, and decreased seizure frequency. We plan to examine the...
CRAD001M2304: A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures
Principal Investigator:
Gipson, Tanjala
This study will investigate the efficacy and safety of everolimus, as adjunctive treatment, in patients ages 2-65 years with Tuberous Sclerosis Complex (TSC) that have refractory partial-onset seizures. The primary objective is to compare the reduction in frequency of partial-onset seizures between...
Features of Self-Injury in TSC
Principal Investigator:
Gipson, Tanjala
Our objective in this study is to look at the feasibility of collecting de-identified data on the topography, severity, and frequency of disruptive behaviors in TSC using an on-line survey.
Reward System in Autism
Principal Investigator:
Goldberg, Melissa
It is a core feature of autism that social interactions appear to be far less rewarding for autistic children compared to typically developing children. Based on this observation, one would predict that children with autism would show different behavioral responses to reinforcers involving social...
An evaluation of differential reinforcement on appropriate toy skills
Principal Investigator:
Gonzalez, Melissa
The purpose of the current study will be to increase the play skills for six children with developmental disabilities, who display limited interest in toys in a free operant setting. It is hypothesized that differential reinforcement of appropriate play behaviors combined with fading will increase...
Program evaluation of caregivers' behavioral workshop
Principal Investigator:
Gonzalez, Melissa
The Pediatric Feeding Disorders Program at Kennedy Krieger Institute provides services aimed at establishing appropriate feeding responses for children who present with a variety of feeding difficulties. Though feeding treatment is the main behavioral concern and reason for treatment, many children...
Translational Analyses of Chronic Aberrant Behavior Across the Lifespan
Principal Investigator:
Hagopian, Louis
The goal of the proposed line of research is to gain a better understanding of the processes that evoke and maintain severe problem behavior (i.e., chronic aberrant behavior) in persons with intellectual and developmental disabilities to improve treatment approaches and outcomes. Despite the...
Behavioral Economics in MR/DD and Behavior Disorders (2)
Principal Investigator:
Hagopian, Louis
Assessments of severe behavior disorders of children with intellectual and developmental disabilities (IDD) have revealed that behavior problems are often maintained by their functional outcomes (or reinforcers). For example, children may gain caregiver attention or escape instructional demands via...
Delineating subtypes of self-injurious behavior maintained by automatic reinforcement
Principal Investigator:
Hagopian, Louis
Self-injurious behavior (SIB) is one of the most serious problems experienced by individuals with intellectual disabilities. The standard of care for the assessment of SIB involves conducting a "functional analysis." This assessment procedure involves observing the individual under several analog...
Evaluation of Assessment and Treatment Data in Individuals with Intellectual/Developmental Disabilities
Principal Investigator:
Hagopian, Louis
The purpose of this retrospective record review is to develop a database to evaluate the efficacy and efficiency of behavior analytic procedures in assessing and treating severe problem behavior.
Pediatric Psychology Consultation-Liaison Services: A Review of Clinical Practice and Patterns
Principal Investigator:
Hendricks, Melissa
Pediatric psychology consultation-liaison (CL) service is the most active form of collaboration between pediatric psychologists and health care specialists. The proposed study is a combination of retrospective record review and a prospective quality improvement/satisfaction survey collecting...
Quantitative mapping of the basal ganglia and related structures in children with dyskinetic cerebral palsy
Principal Investigator:
Hoon, Alexander
Cerebral palsy (CP) refers to a group of motor-impairment syndromes secondary to genetic and acquired disorders of early brain development.  When the underlying disorder affects the basal ganglia (BG) and cortico-striato-thalamic-cortical (CSTC) circuits- from etiologies including acute...
Parental Feedback on the Phelps Center Visual Diagram
Principal Investigator:
Hoon, Alexander
The purpose of this research study is to collect parental feedback on a clinic-developed visual diagram utilized by Dr. Alec Hoon at the Phelps Center for Cerebral Palsy and Developmental Medicine at the Kennedy Krieger Institute. Parental feedback collected from the survey will help improve the...
Evaluation and Development of the IIAM (Important Information About Me) iPad App
Principal Investigator:
Hoon, Alexander
In 2011 a multidisciplinary group at Kennedy Krieger Institute and Johns Hopkins created IIAM (Important Information About Me) as a "voice" for those who cannot "speak”.IIAM is an innovative and comprehensive iPad App under ongoing development to empower children with neurodevelopmental...
IIAM (Important Information About Me): The Ongoing Development of A Novel iPad App
Principal Investigator:
Hoon, Alexander
In 2011 a multidisciplinary group at Kennedy Krieger Institute and Johns Hopkins created IIAM (Important Information About Me) as a "voice" for those who cannot "speak”. IIAM is an innovative and comprehensive iPad app under ongoing development to empower children with neurodevelopmental...
Interrelationships among quantitative measures of white matter injury, sensorimotor function and learning profiles in children with cerebral palsy
Principal Investigator:
Hoon, Alexander
Injury to white matter pathways is strongly associated with the severity of spastic diplegia in children born preterm. In previous research, we have shown that there is preferential injury in sensory pathways than in descending cortiocspinal tracts, and that this injury pattern is associated with...
Comparison of Executive and Adaptive Skills in Individuals Diagnosed with Brain Tumor versus ADHD
Principal Investigator:
Jacobson, Lisa
Neuropsychological assessment and screening is a useful means of identifying cognitive deficits in children and adolescents. Expansion of availability of instruments has now permitted an examination of how these deficits impact "real world" functioning of the individual. Measures of "real world"...
Psychotropic Medication Use at The Center for Child and Family Traumatic Stress: A 4-Year Analysis
Principal Investigator:
Jamora, Mariflor
There have been recent concerns about excessive and inappropriate use of psychotropic medication in foster children, sparked by the recent GAO (Government Accountability Office) report in 2011. Using a de-identified retrospective medical record review, this study proposes to do the following: 1...
Clinical Trials Center Intake, Registry and Questionnaire Database Project
Principal Investigator:
Johnston, MIchael
The Objectives of this protocol are: a. To create a standard intake process for individuals interested in possible participation in clinical trials at the Clinical Trials Unit (CTU), as well as to assess the eligibility of potential research participants for possible participation in IRB-approved...
Student-Centered Behavioral Interventions to Reduce Challenging Behavior in the Classroom
Principal Investigator:
Kahng, SungWoo
Aggressive behavior in young children is a known risk factor for youth violence (CDC, 2011). New methods for preventing youth violence and juvenile delinquency are needed to address this growing concern. Student-centered behavioral interventions (SCBI) provides an empirically-based framework for...
X-Linked Dominant Conradi-Hünermann Syndrome: Clinical and Biochemical Phenotype in Affected Males
Principal Investigator:
Kratz, Lisa
X-Linked Dominant Conradi-Hünermann Syndrome (CPDX2) is a rare disorder of cholesterol biosynthesis caused by a deficiency of 3beta-hydroxysteroid-8, 7-isomerase (sterol isomerase), an enzyme encoded by EBP (emopamil binding protein). Affected heterozygous females present with asymmetric limb...
First Trimester Diagnosis of Smith-Lemli-Opitz Syndrome
Principal Investigator:
Kratz, Lisa
Is sterol analysis of direct chorionic villus material or amniotic fluid an accurate method for first trimester diagnosis of Smith-Lemli-Opitz syndrome?
Risk factors for persistent self injury
Principal Investigator:
Kurtz, Patricia
The purpose of this study is to assess 5 factors believed to be related to the persistence of self-injurious behavior (SIB): parenting practices, child communication, parent depression, parent stress, and lack of social support. Furthermore, we also intend to determine if parenting practices and...
Assessment and treatment of severe problem behavior in individuals with intellectual/developmental disabilities
Principal Investigator:
Kurtz, Patricia
The purpose of the current proposal is to develop a research database comprised of cases treated for severe problem behavior by the Neurobehavioral Inpatient and Outpatient Programs at KKI. The research database will allow for some examination of the relation between assessment procedures and...
Sleep and Cognitive Function after Mild Traumatic Brain Injury in Adolescents
Principal Investigator:
Lam, Janet
After mild traumatic brain injury, many adolescents complain of sleep problems and encephalopathy. This study aims to measure both of these complaints in an objective way to quantify how they are impacted by trauma and the changes that occur in the subsequent recovery process.
Sleep disturbances in neurobehaviorally challenged children with sickle cell disease: A pilot study
Principal Investigator:
Lance, Eboni
This study aims to evaluate sleep in a sample of children, adolescents, and young adults with SCD referred for neurobehavioral evaluation for academic and cognitive difficulties. We will further test the association of sleep disturbance and sleep-disordered breathing with neurobehavioral test...
Autism and the Development of Relational Awareness
Principal Investigator:
Landa, Rebecca
Aims of the proposed research are: to test hypotheses about a deficit in understanding the relationship between objects (e.g., stimulus and reward) in autism, and how this relates to rule learning in autism;  to systematically tease apart and define the developmental progression...
Assessing the Efficacy of a Gaze-Contingent Computer Interaction Game, Targeting Social Communicative Use of Directional Gaze
Principal Investigator:
Landa, Rebecca
This study is being done to examine the impact of an interactive, socially-directed computer game on the use of eye gaze for initiating social interactions in children with autism spectrum disorders (ASD). Eligible families will be randomized to either a 3-week game intervention (n=15) or a no...
Optimizing Social and Communication Outcomes for Young Children with Autism
Principal Investigator:
Landa, Rebecca
The overarching goal of the proposed project is to rigorously test an intervention program for caregivers and their toddlers with autism that is developmentally informed, child-centered and focused on joint attention, a core deficit in young children with autism. Despite the developmental...
Social Skills Intervention for School-aged Children with ASD
Principal Investigator:
Landa, Rebecca
Primary Objective: Our primary objective for this study is to test the effectiveness of two distinct models of social skills group interventions (SKILLS and ENGAGE) with an ethnically and economically diverse group of primary school aged children with an Autism Spectrum Disorder (ASD). The...
Developmental and Augmented Intervention for Facilitating Expressive Language
Principal Investigator:
Landa, Rebecca
Children who do not communicate with spoken words or an augmentative/alternative communication system are an underserved group among those with autism spectrum disorders (ASD). Because caregivers are integral to their children’s development, the treatment will be taught to the caregiver in order...
Parent-Mediated vs. Center Based Intervention for Toddlers with ASD: an RCT
Principal Investigator:
Landa, Rebecca
The major focus of the study is to evaluate the relative impact of two different intervention experiences on social and communication development of minority and underserved toddlers with ASD and their parents. We will to compare the relative effects of a home-based parent-mediated versus a center-...
Training Clinicians in Cognitive Behavior Therapy (CBT) for Children with Autism Spectrum Disorders (ASD)
Principal Investigator:
Landa, Rebecca
Children with high-functioning autism spectrum disorders (ASD) are at greater risk for developing mental health problems, particularly anxiety symptoms. Previous studies have demonstrated reductions in anxiety symptoms in children with ASD following the delivery of specific Cognitive behavioral...
A Comprehensive Approach to Intervention for Toddlers at Risk for ASD
Principal Investigator:
Landa, Rebecca
Research questions are: What are early intervention effects on developmental trajectory and autism symptoms in 14- to 22-month-olds with social and communication delays that indicate high risk for Autism Spectrum Disorders? What are early intervention effects on parents of children...
Early Detection, Intervention, and Neurobiology in Autism: Neurobiology Portion
Principal Investigator:
Landa, Rebecca
This research is being done to learn more about the brain development in children with autism and related disorders. We will be looking at blood samples to examine substances that influence early brain development. There has been some indication that children with autism have unusual levels of...
School-Age Children with Autism with Limited Expressive Language Skills: An Intervention Study
Principal Investigator:
Landa, Rebecca
This project will address a major challenge to the field of autism research: improving expressive communication in children with autism who have reached school age but have not acquired functional spoken language (non-verbal school aged children with autism; NVSACA). This project will provide...
Classroom-Based Early Intervention for Toddlers With Autism
Principal Investigator:
Landa, Rebecca
The proposed project seeks to continue research into the impact of early intervention targeting interpersonal synchrony on the process of social and communication development in toddlers with non-familial autism, which was the focus of our 03-04-29-04: Early Detection, Intervention and Neurobiology...
Early Detection, Intervention and Neurobiology in Autism: Early Intervention Portion
Principal Investigator:
Landa, Rebecca
The overall goal of the study is to counter the effects of increased N-methyl-D-aspartate (NMDA)/glutamate receptors in mutation-positive Rett syndrome (RTT) subjects by use of dextromethorphan (DM), an agent known to block the NMDA receptor channels. The doses of DM used in this study are known to...
NCS Formative Research Project #8 – Development and Validation of Autism Case Confirmation Approaches for Use in the National Children’s Study
Principal Investigator:
Landa, Rebecca
The overarching goal of this proposed project is to assess the criterion validity of three new autism spectrum disorder (ASD) case confirmation instruments against gold standard classification based on the current accepted gold-standard tools - the Autism Diagnosis Observation Schedule (ADOS) and...
Care Navigation for Families of Individuals with Autism Spectrum Disorder
Principal Investigator:
Landa, Rebecca
The proposed study aims to study the effects of a Clinical Services Navigation (CSN) system to improve patient care and family satisfaction for children with a confirmed autism spectrum disorder (ASD) exhibiting co-morbid medical or psychological health conditions and their younger siblings. 
Analyzing Movement of Infants at Risk for Autis
Principal Investigator:
Landa, Rebecca
Research question: 1) Will infants at high-risk for ASD display significantly different motions than infants identified as low-risk for ASD? 2) Can the effect of ASD on motor development be used as an early indicator for identifying infants displaying characteristics of ASD? Using motion...
Development of a Social and Communication Intervention for Preschoolers with Autism
Principal Investigator:
Landa, Rebecca
This is a multiphase study designed to examine the proposed intervention and how it translates for use in authentic educational settings by teachers of preschoolers with ASD. The primary aim of this study is to further develop an early ASD intervention that targets core social and communication...
Autism Treatment Network: Program for the Comprehensive Care and Treatment of Autism
Principal Investigator:
Landa, Rebecca
The purpose of this study is to improve medical treatment of children and adolescents with autism by establishing standards of clinical care based on clinical practice and evidence from clinical outcomes. A core evaluation battery will be given as the standard assessment battery at the Center for...
Relationship Status Among Parents of Children with Autism Spectrum Disorders
Principal Investigator:
Landa, Rebecca
For this study, we will examine the relationship status of parents of children with autism spectrum disorders (ASD). We would like to understand the relationship status of biological parents of children with ASD with one another (single, married/co-habitating, or separated/divorced). In addition,...
Personality Style and Parenting for Fathers of Children with Autism
Principal Investigator:
Landa, Rebecca
Goal: To create a better understanding of the experience of fathers of children with autism spectrum disorders (ASD). Specific Aims: To identify the extent which particular personality dimensions are present among fathers of children with autism To examine the relationships between...
Multisite RCT of Early Intervention for Spoken Communication in Autism
Principal Investigator:
Landa, Rebecca
While significant progress has been made toward identifying effective interventions for preschool-age children with autism (National Research Council, 2001), few scientifically rigorous studies have compared active ingredients of these interventions or examined outcomes focused on core deficits. To...
Validation of a Video-Guided Screening Instrument for Autism Spectrum Disorders
Principal Investigator:
Landa, Rebecca
The overarching goal of this proposed project is to assess the criterion validity of a new autism spectrum disorder (ASD) screening tool by comparing it to a gold standard ASD diagnostic tool known as the ‘Autism Diagnostic Observation Schedule (ADOS)’ paired with the clinical judgment of a...
Autism: Social and Communication Predictors in Siblings
Principal Investigator:
Landa, Rebecca
This is a longitudinal study of Autism Spectrum Disorders (ASD). Six groups will be assessed longitudinally. Group 1: siblings of probands with autism; (Group 2) late talkers having no family history of autism; (Group 3) typically-developing controls; (Group 4) premature controls (for the motor...
Effects of Self-Generated Experiences on Social Cognitive Development in Young Children with Autism
Principal Investigator:
Landa, Rebecca
Sponsored by Autism Speaks -- 7746. This study will test whether a novel treatment for social impairment in young children with autism spectrum disorders (ASD) leads to improvement at behavioral and social information processing levels, addressing Autism Speaks' priority of developing and...
Regulation of Glioblastoma Stem Cells by Kruppel-like Factors
Principal Investigator:
Laterra, John
This research will examine mechanisms by which the family of Kruppel-like factors regulate the behavior, differentiation and tumor initiating ability of human GBM-derived stem cells. We will obtain de-identified GBM-stem cells derived by Dr. Alfredo Quinones from discarded JHH pathological brain...
A Dose-Range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients with Myotonic Dystrophy Type 1
Principal Investigator:
Leung, Doris
A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-Range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients with Myotonic Dystrophy Type 1 This is a phase 1/2a blinded, placebo-controlled, randomized,...
Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
Principal Investigator:
Leung, Doris
This study, supported by the FSH Society, will utilize magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to develop non-invasive, quantitative imaging biomarkers for facioscapulohumeral muscular dystrophy (FSHD), a genetic disorder that causes progressive skeletal muscle...
Bowel and Bladder Continence among Children with Spina Bifida: A Prospective Study
Principal Investigator:
Levey, Eric
The lack of effective bladder and/or bowel control is a significant barrier to the normal social and emotional development of children and adolescents with Spina Bifida. The study proposed in this application will employ a standard, systematic protocol for assessing continence, bowel/bladder...
Pilot Project to Assess Web-Based Family Recruitment for Autism Genetics Studies
Principal Investigator:
Lipkin, Paul
The overall goal of this project is to demonstrate that large-scale DNA collection is feasible and cost-effective. Aim 1: Demonstrate the accuracy of a rapid phenotyping paradigm in which affected status is established by a) parent-report Social Communication Questionnaire (SCQ); b) teacher...
Interactive Autism Network Research Database
Principal Investigator:
Lipkin, Paul
IAN will develop and maintain a national online research database for individuals affected by autism spectrum disorders (ASD) and their family members. This will allow targeted recruitment of individuals based on their profiles to participate in research at KKI and at other research institutes...
Continued Impairment in Adolescents Who Have Outgrown ADHD
Principal Investigator:
Lipkin, Paul
Do adolescents who report that they have "outgrown" childhood Attention-Deficit/ Hyperactivity Disorder (ADHD) (diagnosed before age 10 years of age) continue to have symptoms of ADHD and impairment from this condition?
Outcome of Inclusion in Children with Pragmatic Language Impairment
Principal Investigator:
Lipkin, Paul
To determine if there is a relationship between the level of academic support and reading comprehension level by evaluating the difference in reading comprehension level in third grade in children diagnosed with pragmatic language impairment who are in an inclusion setting and are provided varied...
Developmental Surveillance and Screening Policy Implementation Project
Principal Investigator:
Lipkin, Paul
The Developmental Surveillance and Screening Policy Implementation Project will select 15 pilot primary care pediatric practices to implement the AAP policy statement (anticipated publication of Spring 2006) “Identifying Infants and Young Children with Developmental Disorders in the Medical Home:...
Research Participation Survey
Principal Investigator:
Lipkin, Paul
The Research Participation Survey was designed as an informal operational survey to help the Department of Medical Informatics/IAN Research Project understand what motivates people to participate in autism research, their research interests, and their research experiences. We are now requesting...
Oppositional Disorder, Toilet Training, and Paternal Status
Principal Investigator:
Logue, Dora
Examine the association between age of toilet training, father present in household and later development of oppositional disorder in childhood.
Identification of Barriers and Potential Solutions for Improving Compliance with Mental Health Care Treatment in Children and Adolescents: A Pilot Project
Principal Investigator:
Lopez-Arvizu, Maria Del Carmen
The Department of Psychiatry Clinic at Kennedy Krieger Institute has identified compliance as a major hindrance to mental health care, limiting mental health treatment outcomes and patient satisfaction. Thus, this protocol aims to assess the barriers to mental health care access that underlie non-...
Reading Comprehension: Sources of Difficulty and Neurocognitive Correlates
Principal Investigator:
Mahone, E. Mark
It is well established that successful reading comprehension requires skilled word reading and strong language proficiency, and that word reading deficits are mainly responsible for reading comprehension deficits in some disabled readers at all ages. Beyond the primary grades, however, an...
GRAD Study (Genes, Reading and Dyslexia):Development of a Dyslexia Screening Test
Principal Investigator:
Mahone, E. Mark
This study will examine the relationship between reading associated cognitive competencies and genetic polymorphisms of primarily minority children (those with African or Hispanic heritage) with and without dyslexia, via gene analyses, neurocognitive assessments, and brain imaging.
ADHD in Preschool Girls: Longitudinal Analysis of Neuroimaging and Behavior
Principal Investigator:
Mahone, E. Mark
The overall goal of this project is to characterize the anomalous early development (brain, cognition, and behavior) of girls who present with the behavioral features of Attention Deficit Hyperactivity Disorder (ADHD) in the preschool years, in order to better understand the risk factors, and...
Neuroimaging of Motor Stereotypies in Children with Autism
Principal Investigator:
Mahone, E. Mark
Complex motor stereotypies (i.e., apparently purposeless, rhythmical, repetitive movements) represent one of the core criteria—together with restricted interests, preoccupations, and rigidity—for diagnosis of an autism spectrum disorder in DSM-IV-TR (American Psychiatric Association, 2000; Goldman...
The Effects of ADHD (Beyond Decoding Accuracy) on Reading Fluency and Comprehension
Principal Investigator:
Mahone, E. Mark
The central goal of this project is to understand how ADHD influences processing speed (as it relates to reading speed/fluency), working memory, and reading comprehension. Specifically, we will examine how different components of processing speed and working memory give rise to reading...
ADHD in girls: neuroimaging and executive behavior
Principal Investigator:
Mahone, E. Mark
This study is closed to participant accural and we are currently writing papers. This is a proposal to determine whether ADHD is associated with different brain anomalies/abnormalities in girls and boys. Since girls have not been studied as intensively/extensively as have boys, our focus is on...
Genetic Influences on Sluggish Cognitive Tempo and Reading
Principal Investigator:
Mahone, E. Mark
The overall goal of this pilot study is to examine the relationships between neuropsychological skills and genetic polymorphisms of children ages 8-15 years who display slowed processing speed in the context of behaviorally defined sluggish cognitive tempo (SCT). This is a multisite collaboration...
Neurobehavioral Late-Effects in Pediatric Brain Tumors
Principal Investigator:
Mahone, E. Mark
Radiation Therapy (RT) has been largely responsible for improving the survival rates of children with certain types of brain tumors, although this has come at a cost in terms of the neurodevelopment of these children. Continual modification of RT protocols relies upon accurate risk-benefit data...
Frontostriatal Glutamate in ADHD: Neuropsychological and Behavioral Correlates
Principal Investigator:
Mahone, E. Mark
Sponsored by the National Institutes of Health -- R21MH092693-01. The overall goal of the proposed investigation, "Frontostriatal Glutamate in ADHD: Neuropsychological and Behavioral Correlates" is to investigate the role of frontostriatal glutamatergic metabolism in medication-naïve children with...
Prevalence of Reading Difficulties: 4th-8th Grade
Principal Investigator:
Mahone, E. Mark
The central goal of this project is to estimate the prevalence of various types of reading disability (RD) in the population of upper elementary and middle school students (grades 4 and 8). All data was previously collected in a two-stage probability sampling of public schools throughout the state...
Reading Disability in Grades 3 - 8: Neurocognitive Factors and Behavioral Assessment
Principal Investigator:
Mahone, E. Mark
This project has two purposes: to examine the validity of a response-to-intervention (RTI) approach to identifying children with reading disabilities (RD) who are beyond the initial stages of reading acquisition to describe the cognitive and neurobiological characteristics of children...
Assessment of Neurocognitive Factors in Reading: Grades 4-8
Principal Investigator:
Mahone, E. Mark
The focus of the current research is to examine the extent to and manner in which difficulties in component reading skills (both top down and bottom up), such as decoding accuracy, fluency, and reading comprehension, occur among older children. In doing so, we will also examine the construct of...
CAARE (Creating Advances in Autism through Research Engagement) Project
Principal Investigator:
Menon, Deepa
The CAARE Project will develop and maintain an institutional research database for individuals affected by autism spectrum disorders (ASD) and their family members. This will allow targeted recruitment of individuals based on their profiles to participate in research at KKI. The content of the...
Creating a Pediatric Imaging-Genomics Data Resource
Principal Investigator:
Mostofsky, Stewart
Along with investigators at 8 other sites across the U.S., we will assemble a large, cross-sectional imaging-genomics dataset. The major aim of our proposal is to create a database that is essentially a map depicting the genomic landscape of the developing human brain that can be used as a resource...
Tactile Adaptation in Tourette Syndrome: Probing GABA-Mediated Neuroplasticity
Principal Investigator:
Mostofsky, Stewart
Tourette Syndrome (TS) is a common developmental neuropsychiatric disorder that can have a substantial impact on quality of life. Despite being first described over a century ago, the neurobiology of TS remains poorly understood and therapeutic approaches are modestly effective at best. A deeper...
Quality Assurance of Sequential Finger Tapping Task Data Received from the University of Iowa
Principal Investigator:
Mostofsky, Stewart
This application is being submitted in order to assess with task development of Brain Structure and Function in Juvenile Huntington Disease at Roy J. & Lucille A. Carver College of Medicine. Recruitment, screening, and data collection will not be conducted at Kennedy Krieger Institute or Johns...
Adolescent Changes in Brain and Behavior in Boys and Girls with ADHD
Principal Investigator:
Mostofsky, Stewart
Children with ADHD are at risk for a host of deleterious outcomes including impaired social relations, academic difficulties, criminality, and comorbid psychopathology (substance use, depression, anxiety). Many of these difficulties emerge and exacerbate during adolescence; therefore, it is crucial...
Tourette Syndrome Neuroimaging Consortium Pilot
Principal Investigator:
Mostofsky, Stewart
Important research on Tourette Syndrome (TS) is proceeding along several fronts, from gene-hunting to controlled trials of new treatments. The Tourette Syndrome Association (TSA) has fostered collaborative research in these areas among several centers of excellence.  By laying the groundwork...
Anomalous Motor Physiology in ADHD: Inhibitory and Reward Mechanisms
Principal Investigator:
Mostofsky, Stewart
Attention Deficit Hyperactivity Disorder (ADHD), the most common behavioral diagnosis in children, incurs high medical costs and results in poor academic achievement, adult mental illness, substance abuse, and criminal behavior. An 8-year follow up of the comprehensive Multimodal Treatment of ADHD...
Movement-Based Mindfulness Training for Children with ADHD: A Feasibility Study
Principal Investigator:
Mostofsky, Stewart
Attention Deficit Hyperactivity Disorder (ADHD) has tremendous individual and societal impact. Current standard treatments can provide short-term reductions in symptom severity; however, there remains tremendous need for novel treatment approaches that could remediate the core features of ADHD and...
Neurology of Deficient Response Control in ADHD
Principal Investigator:
Mostofsky, Stewart
Building on evidence established in the initial grant period, the overall goals of the proposed study, “Neurology of Deficient Response Control in ADHD,” are to apply advanced neuroimaging and experimental behavioral methods to understanding the biological basis of ADHD and, as a critical next step...
Motor Skill Learning in Autism
Principal Investigator:
Mostofsky, Stewart
Abnormalities on motor examination have often afforded valuable insights into developmental disorders of the brain; such abnormalities have been well documented in individuals with autism and, in fact, date back to some of the original descriptions of the disorder. Increased insight into the brain...
Anomalus Motor Physiology in ADHD
Principal Investigator:
Mostofsky, Stewart
The hypothesis of this study is that the neuromotor anomalies present in children with ADHD will be associated with deficient intracortical and interhemispheric inhibition of the motor cortex. This hypothesis is consistent with studies suggesting that the central deficit of ADHD is a failure to...
Neurologic basis of inhibitory deficits in ADHD
Principal Investigator:
Mostofsky, Stewart
Prevailing hypotheses implicate deficient response inhibition as one of the fundamental features driving the pathophysiology of ADHD. Results from studies of lesions in animals and humans led many investigators to conclude that response inhibition may be localized specifically to ventral prefrontal...
Tourette Syndrome: Functional Imaging of Motor Activation Pathways in Verbal and Motor Tics
Principal Investigator:
Mostofsky, Stewart
Tourette Syndrome (TS) is a disorder characterized by frequent motor and verbal tics. The exact mechanism of tic generation and the pathophysiology of TS is widely debated but believed to be related to abnormalities within frontal lobe-subcortical circuitry. The goal of this project is to further...
Motor skill learning in autism: assessment and treatment of altered patterns of learning
Principal Investigator:
Mostofsky, Stewart
Detailed study of motor impairments in autism can provide valuable insights into the neural basis of the disorder and help to guide its treatment. Given the developmental nature of autism, during the initial funding period we initiated a research program focused on examining processes underlying...
Human Induced Pluripotent Stem Cells (hiPSC) in Rett syndrome
Principal Investigator:
Naidu, SakkuBai
Skin biopsy will be obtained for fibroblast culture from patients with a known MECP2 mutation as well as skin fibroblasts already present in the KKI’s tissue repository from patients with known mutations in the MECP2 gene. We propose to convert human skin cells to novel cellular types such as...
Pathogenesis of Rett Syndrome
Principal Investigator:
Naidu, SakkuBai
This is an observational study of the natural history and clinical course of Rett Syndrome (RTT), a neurodevelopmental disorder primarily affecting females, caused by mutations in the MeCP2 gene. Our hypothesis is to characterize contributors to the phenotypic variability among mutations in MeCP2...
KKI Weblink for researchers in Rett Syndrome
Principal Investigator:
Naidu, SakkuBai
Creation of a Weblink for Researchers in Rett syndrome to request de-identified data from clinical evaluations collected on our patients with Rett syndrome (RTT) in The Natural History study "Pathogenesis of Rett Syndrome (#03-07-30-10). Participants will not be contacted for any new information....
Dextromethorphan in Rett syndrome
Principal Investigator:
Naidu, SakkuBai
Dr. SakkuBai Naidu of the Kennedy Krieger Institute is initiating a clinical drug trial using dextromethorphan (DM) in Rett Syndrome (RTT) at the Pediatric Clinical Research Unit (PCRU) of the Johns Hopkins Hospital. It has been shown that receptors for a certain brain chemical called glutamine,...
Placebo-Controlled Trial of Dextromethorphan in Rett Syndrome
Principal Investigator:
Naidu, SakkuBai
Dr. Sakkubai Naidu, Principal Investigator, is initiating a double blinded placebo controlledclinical drug trial using dextromethorphan (DM) in Rett Syndrome (RTT), at the PediatricClinical Research Unit (PCRU) of the Johns Hopkins Hospital/Kennedy Krieger Institute, that issponsored by the FDA and...
Perspectives: Cross Sectional Comparison of the Perceptions of Caregivers of Children with Disabilities Regarding Their Own Wellbeing and Availability of Support
Principal Investigator:
O'Connor Leppert, Mary
Our plan is to investigate emotional and physical wellbeing and preparedness for the future among three different cohorts of caregivers: Those with children with intellectual disability Those with children with cerebral palsy Those with children with ADHD (control group...
Database for Preschoolers with Developmental and Behavioral Concerns
Principal Investigator:
O'Connor Leppert, Mary
This study focuses on the development and operation of a clinical database for children (0-5 years of age) with Developmental and Behavioral Concerns attending the preschool interdisciplinary clinic (PIC) at the Kennedy Krieger Institure for routine care. The objective of the database is to capture...
The Medical Evaluation of Autism in Preschoolers: Examining the Discrepancies between Parental and Clinical Impressions
Principal Investigator:
O'Connor Leppert, Mary
The proposed study will examine a clinic population of children being evaluated for early developmental delays in order to investigate how often parents and clinicians differ in their opinions regarding the presence of an ASD diagnosis. This study will further examine which opinion is more likely...
Functional Connectivity ToolKit
Principal Investigator:
Pekar, James
Functional connectivity (FC) is defined as synchrony between neural signals from different locations; collections of regions displaying consistent synchrony are referred to as networks. Spontaneous fluctuations throughout distributed intrinsic networks (INs) modulate BOLD fMRI data acquired from...
Analysis of Genetic Variation in Neurodevelopmental Disorders
Principal Investigator:
Pevsner, Jonathan
We propose to sequence genomic DNA from patients with neurodevelopmental disorders, including autism, to study the nature and extent of genetic variation. To do this, the PI’s laboratory will study genomic DNA samples from two repositories: the Simons Foundation Autism Research Initiative (Sfari)...
Chromosomal abnormalities underlying developmental disabilities
Principal Investigator:
Pevsner, Jonathan
The research question we would like to address is whether children with developmental disabilities have identifiable chromosomal abnormalities. The approach is in four parts: (1) prescreen and recruit patients having developmental disabilities; (2) obtain informed consent and then saliva and...
Using Xbox Kinect based sensory technology to improve the functional mobility by allowing remote monitoring and modification of therapeutic activities for children with Cerebral Palsy in the home
Principal Investigator:
Pidcock, Frank
This is a small fesibility study where Kennedy Krieger will partner with Richard Simpson a rehabilitation engineer to provide the therapeutic and technologic resources for developing and implementing a remote monitoring and tele-therapy gaming system for children with cerebral palsy (CP). Studies...
Developmental Trajectories of Impairments, Associated Health Conditions, and Participation of Children with Cerebral Palsy
Principal Investigator:
Pidcock, Frank
The purpose of this research study is to fill the gaps in fundamental knowledge of Cerebral Palsy by determining how children with cerebral palsy or with gross motor delay change in their balance, range of motion, muscle strength, endurance, health conditions, self-care, and participation in...
Pediatric Constraint-Induced Movement Therapy for Children with Hemiparesis: Outcomes of a Clinical Model
Principal Investigator:
Pidcock, Frank
Does the function of children with hemiparesis improve after treatment involving constraint of the less impaired upper extremity in conjunction with intensive therapies? We will investigate this question by evaluating the function of patients who are receiving standard therapeutic care in our...
Retrospective analysis of risk factors associated with auditory processing disorder: SCAN-3 vs. LiSN-S
Principal Investigator:
Pillion, Joseph
Due to their complexity and variability, disorders involving auditory processing lack a consistent definition and diagnostic criteria. These disorders are characterized by difficulty comprehending of auditory stimuli, despite normal mechanical auditory functioning of the inner and outer ears....
Why parents choose or decline Complementary and Alternative Medicine services in the acute pediatric inpatient rehabilitation setting
Principal Investigator:
Prestwich, Suzanne
Complementary and Alternative Medicine (CAM) usage in the general population is becoming more common as is usage in children. Use in children with chronic illness such as oncology patients, ADHD and Autism have been well documented. However, there is no data about children presenting for acute...
Executive Function and Academic Achievement in Children with a History of Prematurity
Principal Investigator:
Pritchard, Alison
The proposed study will be a retrospective review of previously collected clincal neuropsychological testing data, which will be deidentified for the purpose of this research study. The first objective of this study is to examine specific domains of executive dysfunction that predict poor math...
Construct and Predictive Validity of the Capute Scales
Principal Investigator:
Pritchard, Alison
Primary care physicians (PCPs) are often presented with the responsibility of early identification of developmental delay and other types of disorders, due to their early and ongoing contact with children (Voight et al., 2003). For children born prematurely, early identification is especially...
Outpatient Research Registry
Principal Investigator:
Pritchard, Alison
The primary objective of this protocol is to gather information on Neuropsychology outpatients that can later be accessed for screening purposes (to determine eligibility for research studies conducted within the Neuropsychology Research Lab). Parents and caregivers of patients being evaluated in...
Accommodations for ADHD: Are They Effective in Reducing the Impact of the Disorder?
Principal Investigator:
Pritchard, Alison
One of the most important functions served by a pediatric neuropsychological assessment is the provision of evidence-based recommendations and resources designed to reduce symptoms and improve function and quality of life for youth and their families. In spite of its importance, research into the...
Effect of glycerol trierucate on clinical course of Adrenoleukodystrophy
Principal Investigator:
Raymond, Gerald
Adrenoleukodystrophy is a disorder of very long chain fatty acids secondary to defects in peroxisomal beta oxidation. The accumulation of these fatty acids results in a disorder of the adrenal glands and myelin. A restricted diet and the use of a compound containing glycerol trierucate has been...
Assessing the Impact of Skin Tears on Goal-Directed Therapy Plans Among Individuals with Traumatic Spinal Cord Injury (SCI)
Principal Investigator:
Recio, Albert
To explore how skin tears affect therapy plans in order to promote an optimal wound healing environment and, describe how this modification may influence the achievement of therapy goals among individuals with traumatic SCI.
Collaborative Assessment of Pediatric Transverse Myelitis; Understand, Reveal, Educate. CAPTURE Study
Principal Investigator:
Recio, Albert
Currently, there are no uniform outcome measures to assist in guiding therapies. This multi center study proposes a unique opportunity to examine the outcomes of acute treatment and potentially standardizing outcome measures for a rare neurological disorder. We will utilize a combination of patient...
Virtual Sailing Simulator in Individuals with Spinal Cord Injury
Principal Investigator:
Recio, Albert
The benefits of sport and recreational activities for the disabled are well recognized from the standpoint of health and quality of life. Yet there remains a lack of options for most disabled people, especially the young. Sailing is an attractive sport that can be pursued at recreational and...
Developmental Outcomes in Children with Unilateral and Mild/Moderate Hearing Loss
Principal Investigator:
Reeseman, Jennifer
The primary goal of the present study is to begin to develop a program of research to capture developmental outcomes in children with hearing loss, with a current focus on unilateral (UL) and mild/moderate (MM) hearing loss. The specific aim is to investigate cognitive, educational, and socio-...
Cognitive, Behavioral, and Educational Outcomes in At-Risk Children with Hearing Loss
Principal Investigator:
Reesman, Jennifer
Children with hearing loss are at increased risk for poor cognitive, behavioral and educational outcomes. The present study seeks to identify correlates of poor outcomes and identify predictors via a database review of an at-risk sample presenting for clinical evaluation via the review of de-...
Validation of a Modified Signed Paired Associates Test for Deaf Children
Principal Investigator:
Reesman, Jennifer
There are no published measures available that assess language related memory for children who communicate using American Sign Language (ASL). Most memory measures are created using spoken language, which creates methodological difficulties when translating those measures into ASL. The goal of the...
Utility of the Immediate and Post-Concussion Assessment and Cognitive Testing (ImPACT) test with Deaf and Hard-of-Hearing Adolescents
Principal Investigator:
Reesman, Jennifer
The present research seeks to examine whether the ImPACT test provides an accessible means of assessment for adolescents who are deaf or hard-of-hearing, as this tool has not previously been systematically evaluated for use with this population. The research will be conducted via analysis of two,...
Facial Dysmorphology in Children with Intellectual and Developmental Disabilities
Principal Investigator:
Rooker, Griffin
Recent literature has demonstrated an association between autism spectrum disorders and characteristic dysmorphologies of the face and brain. The current study extends this line of investigation by analyzing the associaton of facial dysmorphology with demographic, behavioral and treatment measures...
Individualizing Learning Arrangements for Children with Intellectual Disabilities
Principal Investigator:
Rooker, Griffin
The frequent and immediate delivery of reinforcers (rewards) following completion of academic tasks and related activities is an integral part of many educational programs for children with developmental and intellectual disabilities. One area of particular interest is the influence of specific...
Delay Discounting in Children with ADHD: Neuroimaging and Behavioral Correlates
Principal Investigator:
Rosch, Keri
My long-term career goal is to establish a research program to improve treatments for ADHD, with an emphasis on doing so through improved understanding of alterations in cognition and motivation in children with ADHD. This program of research has the potential to inform our understanding of the...
Relation of HDL levels with home based Activity Based Restorative Therapies in spinal cord injured adults
Principal Investigator:
Sadowsky, Cristina
Hypothesis: HDL levels increase following home based Activity Based Restorative Therapies (ABRT) The effect of exercise on lipid profile is well documented in the able bodied population. The effect of exercise on lipid profile in individuals with Spinal Cord Injury (SCI) is less clear. The...
The Association Between Lean Body Mass and Functional Outcomes in Individuals with Spinal Cord Injury
Principal Investigator:
Sadowsky, Cristina
What is the correlation between lean body mass (LBM) and functional outcome in individuals with spinal cord injury (SCI)? We plan to determine the association between (LBM) and functional outcomes as measured by the Spinal Cord Independence Measure (SCIM) in individuals with SCI. We plan to conduct...
The Efficacy of Aquatic Locomotor Training Compared to Overground Locomotor Training in Improving Ambulatory Function and Health-Related Quality of Life
Principal Investigator:
Sadowsky, Cristina
The aim of this study is to define and evaluate the efficacy of ALT compared to overground LT in improving ambulatory function and health-related quality of life. We hypothesize, that ALT is capable of producing outcomes that are as good as, or better than, overground LT. ALT may be another tool...
Pediatric Multicenter Evaluation of Notable SCI Outcome Instruments, evaluation of Outcome Instruments when Applied to Children
Principal Investigator:
Sadowsky, Cristina
The overall objective of this research project is to establish pediatric psychometric support (usability, reliability, validity) of outcomes instruments that have been recommended for use in spinal cord injury (SCI) rehabilitation, clinical trials and outcomes research.
SIMS -- Spinal Cord Injury Management System
Principal Investigator:
Sadowsky, Cristina
To define the treatment modality and co-factor variables which influence the response to Activity Based Restorative Therapies (ABRT) in individuals with Spinal Cord Injury (SCI). To define the dose relationship of treatment modalities to the physical and neurological benefits of...
Linking Adult and Pediatric Outcomes Instruments
Principal Investigator:
Sadowsky, Cristina
To validate the estimates used to link the Shriners Hospitals for Children (SHC) pediatric spinal cord injury (SCI) general mobility, manual mobility, power mobility and daily routine computer adaptive tests (CATs) to the adult SCI general mobility, wheeled mobility, self-care, and fine motor CATs...
Relation of Severity of injury with Testosterone profile & osteoporosis in spinal cord injured men
Principal Investigator:
Sadowsky, Cristina
Hypothesis: Higher spinal cord injury neurologic level correlates with decreased testosterone level and increased prevalence of secondary osteoporosis.  Spinal cord injury is more common in young adult men. Because of better care, the life expectancy has increased making them more vulnerable...
Incidence of Osteoporosis in Patients with Chronic Spinal Cord Dysfunction
Principal Investigator:
Sadowsky, Cristina
Research Question: Prevalence of osteoporosis in patients with traumatic and non-traumatic spinal cord injury. Secondary Question: Relationship between bone mass, injury level and grade (ASIA), total and lower extremities motor score, ambulatory status, Functional Electrical Stimulation cycling...
Patterned FES Ergometry of Arm and Shoulder in Individuals with Spinal Cord Injury
Principal Investigator:
Sadowsky, Cristina
Recent evidence suggests that FES can improve certain functions in the Central Nervous System (CNS) following injury or disease; it can enhance motor recovery, change H reflex and flexor reflex excitability [1-3]and enhance sensory function in chronic SCI [4]. Using FES-based therapies in our...
Comorbidities in Children and Adolescents with Seizures
Principal Investigator:
Salorio, Cynthia
This current study will examine symptoms of inattention, hyperactivity, anxiety, and mood problems in a large, clinical sample of children and adolescents with epilepsy. In addition to identifying symptoms commonly reported within the epilepsy sample, comparisons will be made between the epilepsy...
Sedation Strategy and Cognitive Outcome After Critical Illness in Early Childhood: RESTORE-Cognition
Principal Investigator:
Salorio, Cynthia
The purpose of RESTORE-cognition is to determine the relationships between sedative exposure during pediatric critical illness and long-term neurocognitive outcomes. We will assess multiple domains of neurocognitive function 2.5-5 years post-discharge in 500 RESTORE subjects with normal baseline...
ArmAware: a novel device to treat hemiplegia and hemispatial neglect
Principal Investigator:
Salorio, Cynthia
The Investigators have developed a novel therapeutic device called ArmAware, that is designed to be an adjunctive treatment for adults and children with hemiparesis (weakness on one side of the body), by providing stimulation to the affected arm and “reminding” the person to use that arm. We have...
The utilization and efficacy of physical therapy services for individuals with Osteogenesis Imperfecta
Principal Investigator:
Shapiro, Jay
This study is being performed to determine how physical therapy services are being used by individuals with Osteogenesis Imperfecta (OI) and the efficacy of these services for individuals with Osteogenesis Imperfecta. The research study involves completion of a survey about OI and physical therapy...
Factors Determining Failure to Respond to Treatment in Osteogenesis Imperfecta
Principal Investigator:
Shapiro, Jay
Treatment with bisphosphonates is generally effective in increasing bone density and decreasing fracture rate in children with osteogenesis imperfecta. However, adults with osteogenesis imperfecta do not respond to these agents. Also while bisphosphonates administered intravenously are effective in...
Longitudinal Study of Brittle Bone Disease
Principal Investigator:
Shapiro, Jay
The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the natural history, current therapeutic interventions, morbidity, and mortality in people with OI. The research questions are: a. In the longitudinal cohort, what is the prevalence of specific...
A Retrospecitve Evaluation of Bisphosphonate Treatment on Bone Mineral Density and Fracture Incidence in Cerebral Palsy and Spina Bifida Patients
Principal Investigator:
Shapiro, Jay
Children with cerebral palsy and spina bifida, many of whom are non ambulatory, have bone mineral density in the osteopenic/osteoporotic range. These children have an increased risk of fracture. These children have been treated with bisphosphonate. The purpose of this study is review data in the...
Prospective, Multicenter, Single-arm Study to Evaluate Efficacy, Safety and Pharmacokinetics of Denosumab in Children with Osteogenesis Imperfecta
Principal Investigator:
Shapiro, Jay
This is a prospective, multicenter single-arm study in children 2 to 17 years of age with osteogenesis imperfecta (OI). Approximately 150 subjects will be enrolled at 40 institutions including at least 30 subjects less than 7 years of age, and 5 at the KKI. All subjects will receive denosumab 1 mg/...
Assessment of Medical and Surgical Aspects of Cardiovascular Disease in OI
Principal Investigator:
Shapiro, Jay
Current information about cardiovascular disease in OI children or adults has not been systematically evaluated: the real incidence of valvular or aortic disease or atherosclerotic risk or is unknown. Also, information about current cardiac surgical techniques or surgical outcomes applicable to OI...
Retrospective Analysis of Distal Femur and Femur Neck DXA Measurements in Adult Controls and in Patients with Spinal Cord Injury, Cerebral Palsy and Osteogenesis Imperfecta
Principal Investigator:
Shapiro, Jay
Loss of bone mineral density has been a consistent finding in non-weight bearing spinal cord injury (SCI) and cerebral palsy (CP) patients. DXA BMD of the total hip and femur neck and QCT-derived measures of femur bone strength are used to assess fracture risk. Osteogenesis imperfecta is another...
Predicting Cognitive Decline in Adults with Down Syndrome
Principal Investigator:
Silverman, Wayne
The object of this proposal is to predict cognitive decline in adults with Down syndrome (DS) and to ultimately provide a platform for early intervention to prevent the dementia associated with Alzheimer's disease. All research activities will take place at the University of California - Irvine...
Aging and Dementia in Adults with Down Syndrome
Principal Investigator:
Silverman, Wayne
This ongoing NIH supported Program Project will be renewed for another five years, and is programmatically a seamless continuation of my current Exempt protocol (#NA_00002225; "Changes in Functioning Among Mentally Retarded Adults"). The overarching objectives of this program of research will...
The role of Mnbk in DS brain development and aging
Principal Investigator:
Silverman, Wayne
 This collaborative study (headed by Jerzy Wegiel, D.V.D., Ph.D. of the New York State Institute for Basic Research in Developmental Disabilities) focuses on the role of overexpression of Mnbk/Dyrk1A gene product in the development of individuals with Down syndrome. This project will employ...
Changes in Functioning Among Adults with Mental Retardation
Principal Investigator:
Silverman, Wayne
 The major objectives of this program of research are to: (1) study the basic biological mechanisms that underlie Alzheimer disease (AD), focusing explicitly on the adult population with Down Syndrome (DS); (2) quantitatively document the clinical progression in this unique population; and...
Descriptive and Clinical Analysis of Behavioral Services to Enhance Readiness for Transition to Adult Medical Care in Youth with Sickle Cell Disease
Principal Investigator:
Slifer, Keith J.
From a health care systems perspective, one of the main challenges for adolescents with sickle cell disease (SCD) is transitioning from pediatric to adult care. Interdisciplinary transition programs have emerged to promote a smooth transfer of care. The inclusion of behavioral psychology services...
Analysis of and Intervention for Behavioral and Psychological Adjustment and Coping After Pediatric Spinal Cord Injury
Principal Investigator:
Slifer, Keith J.
Sustaining a spinal cord injury (SCI) is a life-altering event, both for youth and their families. Recent innovations in the treatment of pediatric SCI offer the potential for greater restoration of sensory and motor functioning (McDonald & Sadowsky, 2002), but intensive inpatient or outpatient...
Exposure Therapy and Counterconditioning Intervention for Acute Pain Management during Venipuncture in Children with Chronic Conditions: A clinical case series
Principal Investigator:
Slifer, Keith J.
Managing children’s distress during invasive medical procedures, including venipunctures, is challenging for health care providers. The venipuncture procedure is sometimes not well tolerated by children with developmental disabilities due to anxiety about anticipated pain, unfamiliar equipment,...
Psychological and Behavioral Intervention in Pediatric Acquired Brain Injury
Principal Investigator:
Slifer, Keith
Psychological and behavioral interventions are recognized as beneficial components of pediatric rehabilitation; however, there are few empirical studies evaluating the benefits of providing these interventions during rehabilitation after acquired brain injury (Traumatic Brain Injury- TBI,...
Posttraumatic Growth in Pediatric Spinal Cord Injury Survivors
Principal Investigator:
Slifer, Keith
There are two primary objectives to this study. The first is to determine the prevalence of posttraumatic growth and its five factors in a sample of children and adolescents with spinal cord injuries. The second is to identify variables that are potentially correlated with, or predictive of,...
Clinical Assessment of Behavioral Adjustment in Pediatric Patients: A Preliminary Case Series Review
Principal Investigator:
Slifer, Keith J.
In pediatric rehabilitation settings, behavioral consultants see patients with a wide range of medical conditions, mental health diagnoses, and presenting problems. The Pediatric Behavioral Adjustment Scale (PBAS) is a new scale developed to objectively measure child affect and behavior during...
Standardization of the Wechsler Intelligence Scale for Children–Fifth Edition (WISC–V)
Principal Investigator:
Slomine, Beth
The results of this study will be used to create an updated version of one of the most widely-used measures of intelligence for children. We will participate in this research with the test developer, Pearson, as well as other sites from all over the United States and with participants of various...
School And Health Care Transitions To Adulthood For Individuals With Special Health Care Needs In Maryland
Principal Investigator:
Smith, Christopher
The objective of this study is to assess the association between school-based transition planning and health care transition planning for youth with special healthcare needs (YSHCN). The 2011 Maryland Youth Transition Survey, conducted in 2012, will be used to perform a cross-sectional analysis...
Neural basis of impairments in response inhibition and processing speed following pediatric traumatic brain injury
Principal Investigator:
Suskauer, Stacy
Impairments in response inhibition and processing speed developing after pediatric traumatic brain injury (TBI) are common and affect behavioral and academic outcomes. It remains unclear which brain structures, when injured in childhood TBI, are responsible for impaired response inhibition and...
Predictors of functional recovery and description of outcomes after pediatric brain injury
Principal Investigator:
Suskauer, Stacy
Brain dysfunction in children can result from multiple causes, including trauma, hypoxia, diseases of the central nervous system, metabolic disorders, tumors, vascular abnormalities, and congenital neurological disorders. Goals of rehabilitation research in children with brain dysfunction include...
Neural markers of concussion and recovery
Principal Investigator:
Suskauer, Stacy
The goals of this study are to collect preliminary data on neural biomarkers with promise for identifying concussion and predicting and evaluating recovery from concussion in teenagers. We will acquire neuroimaging, neuropsychological, and quantitative sensory data. We will study up to 75 children...
Quantitative Sensory Testing in a Clinical Sample of Children with Concussion
Principal Investigator:
Suskauer, Stacy
Concussion is a diffuse neurometabolic disturbance leading to symptoms in physical, cognitive, emotional and sleep domains. Clinical management of concussion is based on symptom report in conjunction with physical and neurocognitive evaluation. Early imaging data indicate disruptions in neural...
Body Fluid Markers of Pediatric Traumatic Brain Injury
Principal Investigator:
Suskauer, Stacy
The goal of this study is to identify whether blood, urine, and CSF biomarkers are predictive of short-term (1 month) outcome after pediatric traumatic brain injury (TBI). We will obtain blood and urine samples from patients with TBI who, for clinical purposes, already require a blood draw and/or...
Neural Basis of Recovery of Inhibitory Control and Processing Speed After Pediatric TBI
Principal Investigator:
Suskauer, Stacy
Inhibitory control is frequently impaired following pediatric traumatic brain injury (TBI), and deficits in inhibitory control impact a child’s function in school and at home and ultimately their likelihood for successful community integration as an adult. The long-term goal is to understand the...
The Mentoring Practices and Benefits Survey
Principal Investigator:
Swearingen, Tami
The purpose of this multi- centered educational project is to assess the effectiveness of an evidence-based approach to mentoring among inpatient RN's. Specifically the research question posed: Is there a significant pre/post intervention difference in the mentoring practices inventory (MPI) and...
Data Analyses for the Establishment of The Nail Patella Syndrome (NPS) Behavioral Phenotype
Principal Investigator:
Tierney, Elaine
Subjects will not be seen at KKI as part of this study. This study will analyze data collected as part of the previously approved and now closed RPN 00-06-03-05 (entitled, The Behavioral Phenotype of Nail Patella Syndrome). This study will create a database comprised of data/information collected...
Guanfacine in the treatment of hyperactivity and implusiveness in children and adolescents with pervasive development disorders
Principal Investigator:
Tierney, Elaine
This application is a request for transfer of this protocol from paper IRB to e-irb submission review: Previous Application Number (paper based submission): 01-04-06-04 Protocol Title: Guanfacine in the Treatment of Hyperactivity and Impulsiveness in Children and Adolescents with Pervasive...
Methylphenidate in the Treatment of Hyperactivity and Implusiveness in Children and Adolescents with Pervasive Developmental Disorders
Principal Investigator:
Tierney, Elaine
This application is a request for transfer of this protocol from paper IRB to e-irb submission review: This trial has ended. No children will be enrolled or participate in a clinical trial. We request that this protocol remain open until data analyses have been completed. This study...
The Frequency of Smith-Lemli-Opitz Syndrome (SLOS) in the Population with Autism
Principal Investigator:
Tierney, Elaine
The investigators seek to examine specimens gathered by the Autism Genetic Resource Exchange (AGRE) from individuals with autism spectrum disorders (ASD) and their family members (when available), and American Type Culture Collection, other biomaterial repositories, and NIH studies and undergo the...
The Behavioral Phenotype of Smith-Lemli-Opitz-Syndrome and Other Sterol Disorders
Principal Investigator:
Tierney, Elaine
The primary goals of this project are to 1) determine the cognitive, social, and communication abilities and to define the behavioral phenotype of a spectrum of genetically and biochemically well-defined patients with Smith-Lemli-Opitz syndrome (SLOS) and other sterol disorders and 2) examine how...
A Study of Risperidone in Children, Adolescents and Young Adults with Autistic Disorder
Principal Investigator:
Tierney, Elaine
This study seeks to reactivate the following protocols under one new RPN during the post-study analyses phase of these studies: 04-06-03, 99-04-06-02, 99-05-14-01, and 01-04-16-04. Due in part to recent developments regarding the etiology of autism, this study seeks to use archived serum samples (...
Investigation of Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
Principal Investigator:
Tierney, Elaine
This is not a new study, but rather a re-submission of a previously approved study for which IRB approval lapsed. The study is closed to enrollment, but will remain open so that we can 1) continue the follow-up of already enrolled subjects and 2) perform data analyses and manuscript preparation...
Cholesterol in ASD II
Principal Investigator:
Tierney, Elaine
The purpose of this application is to allow for continued data cleaning of the identifiable data collected under terminated study NA_00020928 (PI Tierney; IRB2), including receipt of data from the other study sites and, if applicable, data verification with identifiable source documents.
Glycogen Quantification using MRI
Principal Investigator:
Van Zijl, Peter
The goal of this research is to develop and validate a novel MRI method for imaging glycogen contents of tissue. Glycogen is the main energy storage molecule of the body. Its non-invasive detection in situ in the human would have tremendous utility in the study of sports medicine (muscle glycogen...
Quantitation of the BOLD Effect in Functional MRI
Principal Investigator:
Van Zijl, Peter
The ultimate goal of this project is to gain a complete quantitative understanding of the physiological, physical, and spatial aspects of the blood-oxygen-level-dependent (BOLD) functional MRI (fMRI) approach.
Glucose as contrast agent for MRI
Principal Investigator:
van Zijl, Peter
MRI assessment of tissue perfusion is currently mainly done using GdDTPA contrast enhancement. This contrast agent is FDA approved and used routinely throughout the country in clinical MRI scans of the brain as well as body scans. It has been successfully applied for several decades. Recently,...
Investigation of Emotional Attention and Memory in Children with Anxiety Disorders
Principal Investigator:
Vasa, Roma
This study examines emotional attention and memory in children with anxiety disorders. The data from this study will be compared to data on similar cognitive processes in control children (being collected under protocol NA_00004616. The overall hypothesis is that anxious children will...
Effects of manipulating attention on the neural response to threat stimuli in children with and without anxiety disorders
Principal Investigator:
Vasa, Roma
This study examines whether anxious children exhibit different patterns of brain activation when attending to threatening stimuli compared to healthy control children. 
Investigation of emotional attention and memory in healthy children
Principal Investigator:
Vasa, Roma
This study is collecting data on emotional attention and memory in psychiatrically healthy children. The data from this study will be compared to data on similar cognitive processes in anxious children. The overall hypothesis is that anxious children will preferentially attend to threat vs. neutral...
Psychiatric Crisis among Youth and Transition Age Adults with Autism Spectrum Disorder
Principal Investigator:
Vasa, Roma
The current proposal is a request to assemble a focus group comprised of multidisciplinary clinicians (e.g., psychiatrists, psychologists, social workers) both within and outside the Kennedy Krieger Institute (KKI). The goal of the group will be to discuss and develop two measures. The first is a...
Psychiatric, Behavior, and Cognitive Profiles in Children with Autism Spectrum Disorder
Principal Investigator:
Vasa, Roma
This protocol requests permission to have access to a de-identified research dataset that was collected by the Simons Foundation, a private foundation in New York that focuses on advancing research in the sciences, including the study of autism spectrum disorder (ASD), a chronic neurodevelopmental...
Methylation of Genes in Fetal Alcohol Spectrum Disorders
Principal Investigator:
Vaurio, Rebecca
The goals of this project are as follows: 1. Examine methylation profiles in children diagnosed with FASD compared to children with other psychiatric disorders to evaluate whether there are distinctive patterns of methylation associated with in utero exposure to alcohol. 2. For those differences...
Collection of Data from Muscle Strength Testing
Principal Investigator:
Wagner, Kathryn
The purpose of this protocol is to collect clinical data on muscle strength and function for informed clinical trial designs. The data will be collected from patients with muscular dystrophy during their visits to the Kennedy Krieger Institute Outpatient Center. We will request participants to...
Facioscapulohumeral Disease Biomaterial for Wellstone Core
Principal Investigator:
Wagner, Kathryn R.
An NIH funded, Senatory Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC) has recently been established entitled "Biomarkers for therapy of FSHD (facioscapulohumeral muscular dystrophy)." This multi-institutional MDCRC will be directed by Charles Emerson, Ph.D. at Boston...
An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients with Advanced Stage Duchenne Muscular Dystrophy (Protocol Number 4658-204)
Principal Investigator:
Wagner, Kathryn
This research study is being done to find out if eteplirsen can help people with advanced Duchenne Muscular Dystrophy (DMD). A predecessor trial has been IRB approved (IRB00040305). DMD is caused by a mutation in the gene that makes dystrophin which is important for protecting muscles from stress...
Duchenne Muscular Dystrophy: Double-Blind Randomized Trial to Find The Optimum Steroid Regimen (FOR-DMD)
Principal Investigator:
Wagner, Kathryn R.
This Phase III multi-center, double-blind, parallel group, 36 - 60 month study funded by the NIH will compare three corticosteroid regimens in wide use in DMD: 1) daily prednisone (0.75 mg/kg/day); 2) intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off); and 3) daily deflazacort (...
A Three Dimensional Environment for Skeletal Muscle Stem Cell Transplantation
Principal Investigator:
Wagner, Kathryn R.
The purpose of this collaborative project is to develop and test and injectable scaffold embedded with muscle stem cells and a Pfizer drug to facilitate muscle regeneration. Cell based therapies lag behind the development of other approaches to chronic muscle disease including gene therapy and...
An Open-Label, Multi-Center, 48-Week Study with Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
Principal Investigator:
Wagner, Kathryn
This is an open-label, Phase 3, multi-center, 48-week study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to correction by exon 51 skipping (treated group), with a concurrent control arm...
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Ambulatory Boys with Duchenne Muscular Dystrophy
Principal Investigator:
Wagner, Kathryn
This is a Phase 2 randomized, two-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, pharmacokinetic, and pharmacodynamic of PF-06252616 administered to ambulatory boys diagnosed with DMD ages 6 to <10 years of age. Eligible participants...
Bone health in Facioscapulohumeral muscular dystrophy: A cross-sectional study
Principal Investigator:
Wagner, Kathryn
This is a cross-sectional single visit study to determine bone health in individuals with FSHD. The measures of bone health, including bone mineral density and lean body mass via DEXA scan and blood tests for biomarkers of bone resorption and formation, will be combined with conventional...
Role of acyl-CoA synthetases in lymphangioleiomyomatosis (LAM)
Principal Investigator:
Wakins, Paul A.
LAM is characterized by proliferation of smooth muscle cells in the lung. SLC27A3 is an acyl-CoA synthetase whose activity seems to be increased in rapidly proliferating cells. The fundamental question to be answered is, compared to smooth muscle cells in normal lung, is the expression of SLC27A3...
Assessment of Functional Outcomes of an Interdisciplinary Pediatric Pain Rehabilitation Program
Principal Investigator:
Ward, Cynthia
Pediatric chronic pain can result in functional impairments that may negatively impact ones ability to perform daily activities. Pediatric chronic pain patients may benefit from mutlidisciplinary assessment and recommendations on an outpatient basis, in addition to intensive cognitive behavioral...
Acupressure and Acupuncture as an Intervention with Children with Autism II
Principal Investigator:
Warren, Lana
The purpose of this feasibility study is to see if children with autism spectrum disorder (ASD) will tolerate an acupressure and acupuncture intervention twice weekly over a 3 month period of time that targets regulatory and behavioral functions. It is anticipated that: 1) children with ASD will...
Differences in Behavioral and Adaptive Profiles among Children Diagnosed with Autism Spectrum Disorders, Attention-Deficit/Hyperactivity Disorder, or Anxiety Disorder
Principal Investigator:
Wodka, Ericka
This study is a retrospective review of assessment data from routine psychological or neuropsychological evaluations. The focus of this study is to examine behavioral and adaptive profiles of children evaluated in a neuropsychology clinic and given a diagnosis of ASD, ADHD, or an Anxiety Disorder...
Couples Therapy to Reduce Stress and Conflict with Parents that have a Child with Autism
Principal Investigator:
Wodka, Ericka
To test the efficacy of a couples therapy intervention to reduce stress and conflict in the marital relationship of parents that have a child with autism.
Investigating the neurobehavioral basis of sensory behavior in ASD
Principal Investigator:
Wodka, Ericka
Autism Spectrum Disorder (ASD) is a group of developmental disorders which affects approximately 1% of children. One of the prominent associated symptoms of ASD is abnormal sensory behaviors. While these behaviors limit the child’s and family’s functioning, the underlying causes of these behaviors...
Highly Efficient Conversion Of Human Ips Cells To Dopaminergic Neurons By Synthetic Modified Mrnas
Principal Investigator:
Ying, Mingyao
Ventral midbrain dopaminergic (mDA) neurons derived from human induced pluripotent stem cells (iPSCs) provide a unique cell resource for disease modeling, drug development and cell replacement therapy for Parkinson's disease (PD). Current strategies for generating transplantable iPSC-derived mDA...
Modeling Aggressive Medulloblastoma Using Human Induced Pluripotent Stem Cells
Principal Investigator:
Ying, Mingyao
Brain tumors are the most common cause of childhood oncological death, and medulloblastoma (MB) is the most common malignant pediatric brain tumor. Group 3 MB is characterized by amplification and overexpression of oncogenic transcription factor MYC, herein referred to as MYC-driven MB. Modeling...
The Relationship Between Executive Functioning And Adaptive Functioning In Pediatric Sickle Cell Disease
Principal Investigator:
Zabel, Andrew
Little is known about the relationship between executive functioning in children and adolescents with sickle cell disease (SCD). Similar to other chronic illnesses, children and adolescents with SCD engage in a greater number of daily adaptive tasks, particularly medical self-care tasks, compared...
Ecological validity of neuropsychological testing and screening
Principal Investigator:
Zabel, T. Andrew
Neuropsychological assessment and screening is a useful means of identifying cognitive deficits in children and adolescents. Expansion of availability of instruments has now permitted an examination of how these deficits impact "real world" functioning of the individual. Measures of "real world"...
Evaluating intervention responsiveness in people with multiple sclerosis
Principal Investigator:
Zackowski, Kathleen
Multiple sclerosis (MS) is an inflammatory disease of the central nervous system (CNS). It is characterized by a progressive functional decline although the clinical course of the disease varies considerably from one patient to another, depending in part on the location of plaques. The presently...
Evaluation of data obtained from a Phase III trial to evaluate Lorenzo's oil in adults with X-linked adrenoleukodystrophy
Principal Investigator:
Zackowski, Kathleen
This study will evaluate data collected in a placebo-controlled study of Lorenzo's oil in adult men and women with X-linked adrenoleukodystrophy. This study was originally run from 2005-2008 under Dr. Hugo Moser and Dr. Gerald Raymond. There continues to be aspects of the quantitative sensorimotor...
Mechanisms of Locomotor Recovery in Multiple Sclerosis
Principal Investigator:
Zackowski, Kathleen
The study will test the general hypothesis that, in multiple sclerosis (MS) and following an acute exacerbation, damage of cerebellar white matter tracts (i.e. cerebellar peduncles) will show greater gait ataxia and worse recovery of walking compared to people with damage to motor cortex tracts (i....
Exercise study of function and pathology for women with X-ALD
Principal Investigator:
Zackowski, Kathleen
This study will comprehensively evaluate the corticospinal tract and the dorsal column-medial lemniscal tracts across the brain and cervical spinal cord with the purpose of understanding mechanisms of disability in women with adrenomyeloneuropathy (AMN). We hypothesize that: 1) tract-specific...
Effects of training on caregiver implementation of behavior-analytic skills
Principal Investigator:
Zarcone, Jennifer
This small N study will examine additional effects of our standard of care for caregiver training on the implementation of specific skills with children with severe problem behavior. These skills will be part of their child's treatment plan. A multiple baseline across behaviors will be used to...
Assessment and Treatment of Problem Behavior in Individuals with Intellectual Disabilities
Principal Investigator:
Zarcone, Jennifer
The purpose of this research is to prospectively evaluate clinical data obtained from assessment and treatment procedures for behavior disorders in individuals with intellectual disabilities on the Neurobehavioral Inpatient Unit. We plan to publish our findings

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March is Brain Injury Awareness Month. Learn about the Kennedy Krieger programs that support and treat patients and families with brain injuries.

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