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ClinicalTrials.gov public records Last synced Oct 1, 2026, 5:31 PM EDT

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Showing 25–48 of 474 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Myopathy Clear all
Recruiting Phase 3 Interventional

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

NCT07608432
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Terminated Phase 3 Interventional Results available

A Study of Tadalafil for Duchenne Muscular Dystrophy

NCT01865084
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
7 Years to 14 Years · Male only
Enrollment
331 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
23
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 20 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2019 · Synced Oct 1, 2026, 5:31 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Innovative Ultrasound Technology in Neuromuscular Disease

NCT02104921
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Muscular Dystrophy, Radiculopathy, Myopathy, Polyneuropathy and Mononeuropathies, Trauma Injury, Orthopedic Disorder
Interventions
Not listed
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 90 Years
Enrollment
223 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 11, 2022 · Synced Oct 1, 2026, 5:31 PM EDT
Active, not recruiting No phase listed Observational

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

NCT03458832
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
FSHD-specific functional rating scale, Electrical Impedance Myography
Diagnostic Test · Device
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
324 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
8
States / cities
Los Angeles, California • Kansas City, Kansas • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Terminated No phase listed Observational

Hypertrophic Myopathy in Children

NCT00267462
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 21 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1996
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 7, 2007 · Synced Oct 1, 2026, 5:31 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Investigational Use of Neuromuscular Ultrasound

NCT05237973
View directory record Official record
Conditions
Muscular Dystrophy, Peripheral Neuropathy, Motor Neuron Disorder, Normal Physiology
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 110 Years
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Recruiting Phase 4 Interventional

Once Weekly Infant Corticosteroid Trial for DMD

NCT05412394
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisolone
Drug
Lead sponsor
Anne M. Connolly
Other
Eligibility
1 Month to 30 Months · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
4
States / cities
Chicago, Illinois • Cincinnati, Ohio • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Not listed Phase 1Phase 2 Interventional

Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy

NCT03936894
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Canakinumab Injection [Ilaris]
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
2 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Oct 1, 2026, 5:31 PM EDT
Recruiting Not applicable Interventional

Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients

NCT07766980
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD, Muscular Dystrophy
Interventions
VM100 Nutritional Supplement
Dietary Supplement
Lead sponsor
University of Florida
Other
Eligibility
6 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Recruiting Phase 1 Interventional

Trial of Cell Based Therapy for DMD

NCT06692426
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
MyoPAXon, Tacrolimus
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Completed Phase 1 Interventional

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

NCT03508947
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-210201, Placebo
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
5 Years to 18 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 8, 2019 · Synced Oct 1, 2026, 5:31 PM EDT
No Longer Available No phase listed Expanded access

HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.

NCT05154851
View directory record Official record
Conditions
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
Interventions
HB-adMSCs
Biological
Lead sponsor
Hope Biosciences Research Foundation
Industry
Eligibility
Not listed
U.S. locations
1
States / cities
Sugar Land, Texas
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 1, 2026, 5:31 PM EDT
Completed Phase 2Phase 3 Interventional

Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy

NCT00016653
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Creatine Monohydrate, Glutamine
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
5 Years to 9 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 27, 2011 · Synced Oct 1, 2026, 5:31 PM EDT
Completed Phase 2 Interventional Results available

Weekly Steroids in Muscular Dystrophy

NCT04054375
View directory record Official record
Conditions
Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Prednisone
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Oct 1, 2026, 5:31 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

NCT04851873
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Up to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 9, 2024 · Synced Oct 1, 2026, 5:31 PM EDT
Completed Phase 2 Interventional Results available

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

NCT01462292
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
GSK2402968 3mg/kg/week, GSK2402968 6 mg/kg/week, Placebo to match GSK2402968 3 mg/kg/week, Placebo to match GSK2402968 6 mg/kg/week
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
5 Years and older · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
14
States / cities
Sacramento, California • Stanford, California • Gulf Breeze, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2017 · Synced Oct 1, 2026, 5:31 PM 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 24, 2017 · Synced Oct 1, 2026, 5:31 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-050/NCNP-03, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 15 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Enrolling by invitation No phase listed Observational

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

NCT06606340
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Eteplirsen, Golodirsen, Casimersen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2033
U.S. locations
20
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Oct 1, 2026, 5:31 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

NCT03652259
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy, Type 2E
Interventions
SRP-9003
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 15 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 20, 2026 · Synced Oct 1, 2026, 5:31 PM EDT
Completed Phase 2Phase 3 Interventional

Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

NCT01126697
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions
Coenzyme Q10 and Lisinopril
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
8 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Chicago, Illinois • Charlotte, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2018 · Synced Oct 1, 2026, 5:31 PM 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 1, 2026, 5:31 PM 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 5, 2018 · Synced Oct 1, 2026, 5:31 PM EDT
Not yet recruiting No phase listed Observational

Nemaline Myopathy Clinical Research Network (NM-CTRN)

NCT06774703
View directory record Official record
Conditions
Nemaline Myopathy
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
0 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Palo Alto, California • Bethesda, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 14, 2025 · Synced Oct 1, 2026, 5:31 PM EDT