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ClinicalTrials.gov public records Last synced Oct 6, 2026, 1:04 AM EDT

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Showing 73–96 of 474 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Myopathy Clear all
Terminated Phase 2 Interventional

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
2 Years to 3 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 6, 2026, 1:04 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Nuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral Sclerosis

NCT00023075
View directory record Official record
Conditions
Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 6, 2026, 1:04 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias

NCT02413450
View directory record Official record
Conditions
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 85 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional Results available

A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)

NCT03199469
View directory record Official record
Conditions
X-Linked Myotubular Myopathy
Interventions
Resamirigene bilparvovec
Genetic
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
Up to 5 Years · Male only
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
3
States / cities
Los Angeles, California • Chicago, Illinois • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Completed Phase 1 Interventional

Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids

NCT01388764
View directory record Official record
Conditions
Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
Interventions
L-arginine
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
7 Years to 11 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 3, 2012 · Synced Oct 6, 2026, 1:04 AM EDT
Terminated Phase 2 Interventional

Safety and Efficacy of P-188 NF in DMD Patients

NCT03558958
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
P-188 NF
Drug
Lead sponsor
Phrixus Pharmaceuticals, Inc.
Industry
Eligibility
12 Years to 25 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 11, 2023 · Synced Oct 6, 2026, 1:04 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy

NCT03400852
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
MNK-1411, Placebo
Drug · Other
Lead sponsor
Mallinckrodt ARD LLC
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
5
States / cities
Gulf Breeze, Florida • Atlanta, Georgia • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced Oct 6, 2026, 1:04 AM EDT
Not yet recruiting Not applicable Interventional

iBCI Optimization for Veterans With Paralysis

NCT05470478
View directory record Official record
Conditions
Spinal Cord Injury, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome, Muscular Dystrophy
Interventions
Mobile neural decoding platform (mobile iBCI)
Device
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Home Based Assessments on Participants With DMD

NCT05657938
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Wearable Device, Duchenne Video Assessment (DVA)
Device · Other
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 14, 2023 · Synced Oct 6, 2026, 1:04 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Active, not recruiting Phase 3 Interventional

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

NCT05881408
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
14
States / cities
Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

NCT03769116
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Los Angeles, California • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 6, 2026, 1:04 AM EDT
Recruiting No phase listed Observational

The Duchenne Registry

NCT02069756
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier, Dystrophinopathy Female Carrier
Interventions
Not listed
Lead sponsor
The Duchenne Registry
Other
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2047
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Active, not recruiting No phase listed Observational

LGMD R1 Natural History Study

NCT05618080
View directory record Official record
Conditions
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type R1, LGMD2A
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
12 Years to 50 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
9
States / cities
Orange, California • Aurora, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Terminated Phase 1 Interventional Results available

Spironolactone Versus Prednisolone in DMD

NCT03777319
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Spironolactone, Prednisolone
Drug
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years to 7 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
4
States / cities
Iowa City, Iowa • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2023 · Synced Oct 6, 2026, 1:04 AM EDT
Terminated Phase 1 Interventional

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)

NCT05906251
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy
Interventions
SRP-6004
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Oct 6, 2026, 1:04 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Nitric Oxide Production in MELAS Syndrome

NCT01339494
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Arginine and citrulline supplementations
Dietary Supplement
Lead sponsor
Baylor College of Medicine
Other
Eligibility
3 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2016 · Synced Oct 6, 2026, 1:04 AM EDT
Terminated Phase 2 Interventional Results available

Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy

NCT01168908
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Sildenafil
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 6, 2026, 1:04 AM EDT
Completed Phase 3 Interventional Results available

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

NCT02851797
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
givinostat, placebo
Drug
Lead sponsor
Italfarmaco
Industry
Eligibility
6 Years to 17 Years · Male only
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
13
States / cities
Davis, California • San Diego, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2023 · Synced Oct 6, 2026, 1:04 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension

NCT02531217
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 18, 2023 · Synced Oct 6, 2026, 1:04 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Urinary Titin Biomarker in DMD

NCT07332013
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
Interventions
Descending stair walk
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 10 Years · Male only
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 6, 2026, 1:04 AM EDT
Recruiting Phase 1Phase 2 Interventional

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

NCT04906460
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-N531
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
2
States / cities
Little Rock, Arkansas • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 6, 2026, 1:04 AM EDT
Terminated Phase 2 Interventional Results available

Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities

NCT04572893
View directory record Official record
Conditions
Primary Familial Dilated Cardiomyopathy
Interventions
danicamtiv
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years to 80 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
12
States / cities
La Jolla, California • Washington D.C., District of Columbia • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated May 21, 2025 · Synced Oct 6, 2026, 1:04 AM EDT
Completed Phase 1 Interventional

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

NCT06204809
View directory record Official record
Conditions
Myotonic Dystrophy 1
Interventions
PGN-EDODM1 for infusion, Placebo
Drug · Other
Lead sponsor
PepGen Inc
Industry
Eligibility
18 Years to 60 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
7
States / cities
Irvine, California • Palo Alto, California • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2026 · Synced Oct 6, 2026, 1:04 AM EDT