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Clinical Research Projects

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...
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....
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...
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...
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...
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...
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...
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...
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/...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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),...
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-...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...

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