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Conditions: Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
Interventions: Not listed
Lead sponsor: Virginia Commonwealth University; Other
Eligibility: 6 Years and older · Male only
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 9
States / cities: Orange, California • Aurora, Colorado • Iowa City, Iowa + 6 more

Conditions: Muscular Dystrophy
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 8 Years to 100 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Congenital Muscular Dystrophy
Interventions: Omigapil; Drug
Lead sponsor: Santhera Pharmaceuticals; Industry
Eligibility: 5 Years to 16 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Developmental Disability, Intellectual Disability, Autism Spectrum Disorder, Congenital Anomaly, Fragile X Syndrome, Facioscapulohumeral Muscular Dystrophy 1
Interventions: Standard of care genetic testing group; Other
Lead sponsor: Bionano Genomics; Industry
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 8
States / cities: Atlanta, Georgia • Augusta, Georgia • Iowa City, Iowa + 5 more

Conditions: Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children, Vasodilation, Exercise, DMD
Interventions: Tadalafil, Placebo, Exercise Training; Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 6 Years and older · Male only
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions: Not listed
Lead sponsor: Cooperative International Neuromuscular Research Group; Network
Eligibility: 8 Years to 18 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 2
States / cities: Washington D.C., District of Columbia • Pittsburgh, Pennsylvania

Conditions: Duchenne Muscular Dystrophy
Interventions: Deflazacort; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 4 Years and older · Male only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 4
States / cities: Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 more

Conditions: Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
Interventions: Zeleciment Rostudirsen (DYNE-251), Placebo; Drug
Lead sponsor: Dyne Therapeutics; Industry
Eligibility: 4 Years to 18 Years · Male only
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 1
States / cities: Hillsborough, North Carolina

Conditions: Stress
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years and older
Enrollment: 228 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2017
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions: Neurosleeve; Device
Lead sponsor: Thomas Jefferson University; Other
Eligibility: 4 Years and older
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2026
U.S. locations: 2
States / cities: Wilmington, Delaware • Philadelphia, Pennsylvania

Conditions: Myotonic Dystrophy 1
Interventions: Not listed
Lead sponsor: Stanford University; Other
Eligibility: 12 Years to 90 Years
Enrollment: 125 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Congenital Muscular Dystrophy
Interventions: Hyperinsufflation therapy; Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 5 Years to 20 Years
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 2
States / cities: Cincinnati, Ohio • Philadelphia, Pennsylvania

Conditions: Incomplete Spinal Cord Injury, Muscular Dystrophy, Spina Bifida, Cerebral Palsy
Interventions: EA-KAFO; Device
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: 5 Years to 100 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2025
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Duchenne Muscular Dystrophy
Interventions: Prednisolone; Drug
Lead sponsor: Washington University School of Medicine; Other
Eligibility: 1 Month to 30 Months · Male only
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 6
States / cities: Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more

Conditions: Duchenne Muscular Dystrophy
Interventions: Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5, Placebo; Drug
Lead sponsor: Edgewise Therapeutics, Inc.; Industry
Eligibility: 4 Years to 9 Years · Male only
Enrollment: 76 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 14
States / cities: Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 11 more

Conditions: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions: Not listed
Lead sponsor: Cooperative International Neuromuscular Research Group; Network
Eligibility: 8 Years to 18 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2012
U.S. locations: 5
States / cities: Sacramento, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more

Conditions: Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Incomplete Spinal Cord Injury
Interventions: EA-KAFO; Device
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: 3 Years to 17 Years
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Muscle Weakness, Problems Moving Their Arms and Legs
Interventions: Extension assist knee ankle foot orthosis (EA-KAFO); Device
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: 5 Years to 25 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Nonsene Mutation Duchenne Muscular Dystrophy
Interventions: Ataluren; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 6 Months to 2 Years · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Duchenne Muscular Dystrophy
Interventions: Deflazacort; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 4 Years to 16 Years · Male only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 4
States / cities: Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 more

Conditions: Duchenne Muscular Dystrophy
Interventions: SGT-001; Genetic
Lead sponsor: Solid Biosciences Inc.; Industry
Eligibility: 4 Years to 17 Years · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 2
States / cities: Los Angeles, California • Gainesville, Florida

Conditions: LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
Interventions: Not listed
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: Up to 5 Years
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 14
States / cities: Los Angeles, California • Stanford, California • Chicago, Illinois + 11 more

Conditions: Duchenne Muscular Dystrophy
Interventions: Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Placebo; Drug
Lead sponsor: Edgewise Therapeutics, Inc.; Industry
Eligibility: 6 Years to 17 Years · Male only
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 7
States / cities: Los Angeles, California • Sacramento, California • Gainesville, Florida + 4 more

Conditions: Duchenne Muscular Dystrophy (DMD)
Interventions: Not listed
Lead sponsor: University of Rochester; Other
Eligibility: 0 Days to 3 Years · Male only
Enrollment: 105 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 1
States / cities: Rochester, New York

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Defining Endpoints in Becker Muscular Dystrophy

Heart Imaging in Children With Muscular Dystrophy

Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)

Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort

Vasodilator and Exercise Study for DMD (VASO-REx)

Cardiac Magnetic Resonance in Children With Muscular Dystrophy

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

Where Does Hope Fit In? The Relationship Between Hope, Uncertainty, and Coping Efficacy in Mothers of Children With Duchenne/Becker Muscular Dystrophy

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy

A Study to Test Lung Stretch Therapy (Hyperinsufflation) in Children With Collagen VI Muscular Dystrophy

Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

Cardiac Outcome Measures in Children With Muscular Dystrophy

Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders

Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults

A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy