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Showing 1–24 of 402 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Muscular Dystrophies Clear all
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 Jun 7, 2026, 12:23 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

NCT00264888
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PTC124
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jan 13, 2009 · Synced Jun 7, 2026, 12:23 PM EDT
Completed No phase listed Observational

A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD

NCT01437345
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
7
States / cities
Sacramento, California • Washington D.C., District of Columbia • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 10, 2017 · Synced Jun 7, 2026, 12:23 PM EDT
Terminated Phase 3 Interventional

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

NCT01803412
View directory record Official record
Conditions
Muscular Dystrophies
Interventions
Drisapersen
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
5 Years and older · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
10
States / cities
Sacramento, California • Tampa, Florida • Iowa City, Iowa + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2018 · Synced Jun 7, 2026, 12:23 PM 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 Jun 7, 2026, 12:23 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

NCT05524883
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
DYNE-251, Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
4 Years to 16 Years · Male only
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
11
States / cities
La Jolla, California • Los Angeles, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2025 · Synced Jun 7, 2026, 12:23 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
SAR446268
Biological
Lead sponsor
Sanofi
Industry
Eligibility
10 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
4
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jun 7, 2026, 12:23 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

NCT03433807
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years and older
U.S. locations
12
States / cities
Phoenix, Arizona • New Haven, Connecticut • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 20, 2023 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Phase 2 Interventional Results available

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03038399
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day, Vamorolone 6.0 mg/day/day
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 19, 2021 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Not applicable Interventional

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

NCT01836627
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 25, 2016 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02760264
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day, Vamorolone 6.0 mg/kg/day
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 6 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 1, 2019 · Synced Jun 7, 2026, 12:23 PM EDT
Terminated No phase listed Observational

Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

NCT03867435
View directory record Official record
Conditions
Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
11 Years to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Jun 7, 2026, 12:23 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 7, 2019 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

NCT00577577
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
rhIGF-I/rhIGFBP-3, placebo
Drug
Lead sponsor
Insmed Incorporated
Industry
Eligibility
21 Years to 65 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
12
States / cities
Orange, California • Sacramento, California • Kansas City, Kansas + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 5, 2022 · Synced Jun 7, 2026, 12:23 PM EDT
Terminated Early Phase 1 Interventional Accepts healthy volunteers

The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

NCT02653833
View directory record Official record
Conditions
Muscular Dystrophy
Interventions
Tadalafil 20 MG, beetroot juice extract
Drug · Other
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 45 Years · Male only
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Phase 1 Interventional

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

NCT02295748
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Jun 7, 2026, 12:23 PM 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 20, 2025 · Synced Jun 7, 2026, 12:23 PM EDT
Active, not recruiting Phase 2 Interventional

Study of BBP-418 in Patients With LGMD2I

NCT04800874
View directory record Official record
Conditions
LGMD2I
Interventions
BBP-418
Drug
Lead sponsor
ML Bio Solutions, Inc.
Industry
Eligibility
12 Years to 55 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated May 7, 2025 · Synced Jun 7, 2026, 12:23 PM EDT
Completed No phase listed Observational

Multicenter Observational Study of Myotonic Dystrophy Type 1

NCT02308657
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 70 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
6
States / cities
Stanford, California • Gainesville, Florida • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 25, 2017 · Synced Jun 7, 2026, 12:23 PM EDT
Terminated Phase 2 Interventional

HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

NCT02525302
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
HT-100
Drug
Lead sponsor
Akashi Therapeutics
Industry
Eligibility
6 Years to 20 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
5
States / cities
Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2019 · Synced Jun 7, 2026, 12:23 PM EDT
Recruiting Phase 1 Interventional

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

NCT06224660
View directory record Official record
Conditions
DMD-Associated Dilated Cardiomyopathy
Interventions
SRD-001
Genetic
Lead sponsor
Sardocor Corp.
Industry
Eligibility
18 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Kansas City, Kansas • Cincinnati, Ohio • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 26, 2025 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Not applicable Interventional Results available

Cardiomyopathy in DMD: Lisinopril vs. Losartan

NCT01982695
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Cardiomyopathy
Interventions
Losartan, Lisinopril
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
6
States / cities
Davis, California • Kansas City, Kansas • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2017 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Phase 1Phase 2 Interventional

Pentoxifylline in Duchenne Muscular Dystrophy

NCT00102453
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Pentoxifylline
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years to 7 Years · Male only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Rochester, Minnesota • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2011 · Synced Jun 7, 2026, 12:23 PM EDT
Completed Phase 2 Interventional Results available

Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

NCT01396239
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
AVI-4658 (Eteplirsen), Placebo
Drug · Other
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 13 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 29, 2020 · Synced Jun 7, 2026, 12:23 PM EDT