Current Clinical Trials

Currently Recruiting Trials

A Phase 4, Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study of the Efficacy and Safety of SPN 812 in Preschool-Age Children (4 to 5 years old) with Attention-Deficit/Hyperactivity Disorder (ADHD)

Description:  This research study is being conducted to evaluate the efficacy of SPN-812 100mg compared to placebo on ADHD symptoms in preschool-age children with DSM-IV-TR. Eligibility includes, but is not limited to children ages 4yrs 0 months and 5yrs 9 months with a primary diagnosis of ADHD (children in the process of being diagnosed can also qualify), is participating in a structured group activity (e.g., preschool, kindergarten, sports, Sunday school or childcare program). The study duration is 10 weeks which consists of 8 onsite visits and includes ECGs and laboratory tests.

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

Description: The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD.

A Long-term Observational Study Evaluating Exon-Skipping Therapies in Patients with Duchenne Muscular Dystrophy (EVOLVE)

Description: This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.

Characterization of the Natural History of Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation

Description: In this study, we will conduct retrospective chart and imaging reviews and prospective longitudinal virtual assessments of individuals with LBSL.

Cardiopulmonary Outcomes in Osteogenesis Imperfecta (BBD7708)

Description: Although OI is typically characterized as a disease of the bone, it is perhaps more accurately described as a connective tissue disorder. Type I collagen is a major constituent of lung connective tissue. Respiratory insufficiency is the leading cause of death in patients with OI. Thus, it is important and necessary to understand the etiology of the restrictive pulmonary physiology in the OI population.

The Balance Study – Bipolar Disorder (Allergan)

Description: Bipolar depression can change the way a child or teen interacts with the world around them. If you or your child have bipolar depression, we are inviting you to take part in the Balance Study for children and teens, aged 10 to 17 years of age, who have been struggling with bipolar depression. The purpose of this study is to evaluate an investigational drug for bipolar depression in children and teens. The Balance Study will enroll about 380 children and teens with bipolar depression at participating study centers throughout North America and Europe.  

Forward-March Fragile X Study

Description:  FORWARD-MARCH is the next step following a highly successful research study called FORWARD (Fragile X Online Registry With Accessible Research Database) that was started in 2012. The FORWARD study created the largest database of information on fragile X syndrome (FXS) in the United States. Data from the FORWARD study is being used by researchers to learn about the lives of people with FXS, including medication use, behaviors, and development over time, and share findings with other researchers and clinicians.

Amgen Osteogenesis Imperfecta Study

Description:  A Phase 3, Open-label, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Romosozumab Compared with Bisphosphates in Children and Adolescents with Osteogenesis Imperfecta

Multimodal brain imaging of the neural effects of methylphenidate in patients with ADHD

Description: This research study is being conducted to evaluate how the study drug, methylphenidate, affects brain activity and brain chemistry in righthanded children ages 6-17 years with ADHD. The study consists of three onsite visits and involves neuropsychological testing along with functional MRI and magnetic resonance spectroscopy (MRS) scans after a single dose of each treatment (i.e., placebo, low dose of methylphenidate, and higher dose). Eligible participants may earn up to $300 for completing the study.

Cerebral Adrenoleukodystrophy (CALYX) 

Description: A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects with Cerebral Adrenoleukodystrophy 

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative clinical Trials Network (GLIA-CTN)

Description: The aim of this study is to create a multi-site biorepository of medical records and bio samples from patients affected with a leukodystrophy and age & sex matched controls.

Genetic Causes of Intellectual Disability 

Description: The aim of this study is to obtain plasma from both affected and healthy individuals to test for metabolomics to discover a biomarker for AMN. 

View study flyer

Kennedy Krieger Institute Research Video Repository

This study is currently recruiting.

Study Description: This research aims to collect and store videos of patients with neurological conditions. These videos will be referenced by clinicians and approved staff as a resource for future education and research.

The Neuronal Ceroid Lipofuscinoses Natural History Study

This study is currently recruiting.

Study Description: This is a natural history study of Neuronal Ceroid Lipofuscinosis (NCL) disorders, including all known forms of NCL (e.g., CLN1 disease, CLN2 disease, etc.). The goals of the study are to learn more about the genetic changes that cause NCL disorders, the typical ages when symptoms start, how symptoms progress, and the ways that mobility (walking) is affected.

Active, but not enrolling:

Ultragenyx Study to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta

Description: An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and Phase 3 Double-Blind, Placebo-Controlled Phase to Assess The Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta (ULTRAGENYX)

Rare Disease Clinical Research Network Brittle Bone Disease Consortium Longitudinal Study of Osteogenesis Imperfecta (RDCRN #7701)

Description: Rare Disease Clinical Research Network Brittle Bone Disease Consortium Longitudinal Study of Osteogenesis Imperfecta (RDCRN #7701).

A Long-term Safety Study to Assess the Potential for Chronic Liver Injury in Participants Treated with Epidiolex (Cannabidiol) Oral Solution (GWEP19022)

Description: This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.

Prospective study of sensory motor function testing and Magnetic Resonance Imaging of brain and spinal cord in adrenoleukodystrophy and related leukodystrophies

Description: Prospective study of sensory motor function testing and Magnetic Resonance Imaging of brain and spinal cord in adrenoleukodystrophy and related leukodystrophies

Neuronal Ceroid Lipofuscinosis (NCL) Natural History Study

Description: Neuronal Ceroid Lipofuscinosis (NCL) Natural History Study

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

Description: Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy (CANYON)

Description: CANYON is a double-blind, randomized, placebo-controlled design to investigate the effect of sevasemten on the safety, pharmacokinetics, biomarkers, and functional measures. Approximately 32 adults and 18 adolescents with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, a 12-month Treatment period, followed by a 4-week follow-up period.

A Phase II Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants with Facioscapulohumeral Muscular Dystrophy

Description:  The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in ambulant adult participants with facioscapulohumeral muscular dystrophy (FSHD).

A Phase 3 Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 2I (FORTIFY)

Description: This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile. This will encompass the significant majority of existing diagnosed patients based upon the established epidemiology of the disease.

Contact Us

1741 Ashland Avenue
Baltimore, MD 21205
Phone: (443) 923-3850
Email: ResearchTrials@KennedyKrieger.org