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Showing 1–24 of 410 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Muscular Dystrophies Clear all
Terminated Phase 3 Interventional Results available

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

NCT02090959
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years to 15 Years · Male only
Enrollment
219 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
21
States / cities
Los Angeles, California • Sacramento, California • Stanford, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2020 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Tadalafil in Becker Muscular Dystrophy

NCT01070511
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 19, 2013 · Synced Sep 24, 2026, 10:13 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy

NCT02269865
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
8 Years to 17 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 20, 2014 · Synced Sep 24, 2026, 10:13 AM EDT
Recruiting No phase listed Observational

Clinical Trial Readiness for the Dystroglycanopathies

NCT00313677
View directory record Official record
Conditions
Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Katherine Mathews
Other
Eligibility
Not listed
Enrollment
190 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2030
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Phase 1 Interventional

rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies

NCT02710500
View directory record Official record
Conditions
Dysferlinopathy
Interventions
rAAVrh74.MHCK7.DYSF.DV
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 12, 2021 · Synced Sep 24, 2026, 10:13 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT06138639
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
0 Years to 17 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
12
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Sep 24, 2026, 10:13 AM EDT
Recruiting No phase listed Observational

Long-Term Development of Muscular Dystrophy Outcome Assessments

NCT05989620
View directory record Official record
Conditions
LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 50 Years
Enrollment
1,000 participants
Timeline
2023 – 2029
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 17, 2025 · Synced Sep 24, 2026, 10:13 AM EDT
Completed No phase listed Observational

Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

NCT01506518
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
5 Years to 13 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 1, 2021 · Synced Sep 24, 2026, 10:13 AM EDT
Recruiting Phase 3 Interventional

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

NCT07486934
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy, Steinert Disease, Steinert
Interventions
zeleciment basivarsen (DYNE-101), Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
16 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
10
States / cities
La Jolla, California • Los Angeles, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 24, 2026, 10:13 AM EDT
Terminated Phase 2 Interventional

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

NCT04004065
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vesleteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
11
States / cities
Sacramento, California • Farmington, Connecticut • Gulf Breeze, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2025 · Synced Sep 24, 2026, 10:13 AM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 24, 2026, 10:13 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Muscle Tissue Bank for Muscular Dystrophy

NCT01950897
View directory record Official record
Conditions
Muscular Dystrophies, Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
6 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Sep 24, 2026, 10:13 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 25, 2019 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Phase 1 Interventional

Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy

NCT02376816
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.micro-Dystrophin
Biological
Lead sponsor
Jerry R. Mendell
Other
Eligibility
7 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 23, 2017 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Feasibility of Non-invasive Dried Blood Sampling

NCT05492734
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
EDG-5506
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
19 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
Lincoln, Nebraska
Source: ClinicalTrials.gov public record
Updated Sep 1, 2022 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Not applicable Interventional Results available

ECoG Direct Brain Interface for Individuals With Upper Limb Paralysis

NCT01393444
View directory record Official record
Conditions
Tetraplegia, Spinal Cord Injury, Brachial Plexus Injury, Muscular Dystrophy, ALS, Brainstem Stroke
Interventions
Implantation of ECoG sensors on the brain surface
Device
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 70 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 11, 2016 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy

NCT02239224
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Placebo, ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Rochester, New York • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 10, 2021 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD

NCT02834650
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test, Heart Rate, Pulmonary Function Test, Genetic Testing, Repeat MRI scan
Other
Lead sponsor
Stanford University
Other
Eligibility
7 Years to 21 Years · Male only
Enrollment
89 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2022
U.S. locations
3
States / cities
Los Angeles, California • Orange, California • Stanford, California
Source: ClinicalTrials.gov public record
Updated May 9, 2022 · Synced Sep 24, 2026, 10:13 AM EDT
Completed No phase listed Observational

The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

NCT01182324
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Sep 24, 2026, 10:13 AM EDT
Completed No phase listed Observational

Evaluation and Diagnosis of People With Pain and Fatigue Syndromes

NCT00677157
View directory record Official record
Conditions
Fatigue
Interventions
Not listed
Lead sponsor
National Institute of Nursing Research (NINR)
NIH
Eligibility
18 Years and older
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 21, 2020 · Synced Sep 24, 2026, 10:13 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT03836300
View directory record Official record
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 24, 2026, 10:13 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls

NCT01523964
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Testing with EIM
Other
Lead sponsor
Dart Therapeutics. LLC
Other
Eligibility
3 Years to 12 Years · Male only
Enrollment
92 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012
U.S. locations
6
States / cities
Aurora, Colorado • Boston, Massachusetts • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2013 · Synced Sep 24, 2026, 10:13 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 17, 2023 · Synced Sep 24, 2026, 10:13 AM EDT
Enrolling by invitation Phase 3 Interventional

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07008469
View directory record Official record
Conditions
Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
Interventions
Del-desiran (AOC 1001)
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
19
States / cities
Los Angeles, California • Stanford, California • Denver, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 24, 2026, 10:13 AM EDT