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ClinicalTrials.gov public records Last synced Oct 13, 2026, 2:41 PM EDT

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Showing 169–192 of 237 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Duchenne Muscular Dystrophy (DMD) Clear all
Completed Phase 3 Interventional Results available

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

NCT03703882
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Edasalonexent, Placebo
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
24
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2022 · Synced Oct 13, 2026, 2:41 PM EDT
Not listed No phase listed Observational

Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

NCT01539772
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years and older · Male only
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
13
States / cities
Sacramento, California • Washington D.C., District of Columbia • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2018 · Synced Oct 13, 2026, 2:41 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Burden of Access in Duchenne Muscular Dystrophy in the US

NCT03951675
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 99 Years
Enrollment
57 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 6, 2020 · Synced Oct 13, 2026, 2:41 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

NCT06839469
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 13, 2026, 2:41 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

NCT02964377
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
(+)- Epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
8 Years to 17 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 21, 2021 · Synced Oct 13, 2026, 2:41 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00847379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
15
States / cities
Sacramento, California • Aurora, Colorado • Gulf Breeze, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2020 · Synced Oct 13, 2026, 2:41 PM EDT
Available No phase listed Expanded access

Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

NCT07250737
View directory record Official record
Conditions
Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Dystrophies, Muscular Dystrophy, Duchenne
Interventions
delpacibart zotadirsen
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
2 Years and older · Male only
U.S. locations
22
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Irvine, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 13, 2026, 2:41 PM EDT
Terminated Phase 3 Interventional Results available

Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)

NCT04371666
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Pamrevlumab, Placebo, Corticosteroids
Drug
Lead sponsor
Kyntra Bio
Industry
Eligibility
12 Years and older · Male only
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
24
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 21 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2024 · Synced Oct 13, 2026, 2:41 PM EDT
Not listed Phase 1 Interventional

Treatment of a Single Patient With CRD-TMH-001

NCT05514249
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
CRD-TMH-001
Drug
Lead sponsor
Cure Rare Disease, Inc
Other
Eligibility
18 Years to 28 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 1, 2022 · Synced Oct 13, 2026, 2:41 PM EDT
Completed Phase 2 Interventional Results available

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02740972
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-065/NCNP-01, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 9 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Sacramento, California • Gainesville, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2021 · Synced Oct 13, 2026, 2:41 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03439670
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone, Prednisone, Placebo
Drug · Other
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
10
States / cities
Davis, California • Los Angeles, California • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2023 · Synced Oct 13, 2026, 2:41 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 27, 2011 · Synced Oct 13, 2026, 2:41 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT03648827
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
2 Years to 7 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
9
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 5, 2022 · Synced Oct 13, 2026, 2:41 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

NCT03992430
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 13 Years · Male only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
3
States / cities
Birmingham, Alabama • Gainesville, Florida • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 13, 2026, 2:41 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

NCT00468832
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
2 Years to 30 Years · Male only
Enrollment
551 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2019
U.S. locations
11
States / cities
Sacramento, California • Washington D.C., District of Columbia • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2016 · Synced Oct 13, 2026, 2:41 PM EDT
Completed Phase 1 Interventional

Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy

NCT04386304
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(+)-Epicatechin
Drug
Lead sponsor
Epirium Bio Inc.
Industry
Eligibility
16 Years to 59 Years · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
3
States / cities
Los Angeles, California • Sacramento, California • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 23, 2022 · Synced Oct 13, 2026, 2:41 PM EDT
Completed Phase 3 Interventional Results available

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

NCT05096221
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 13, 2026, 2:41 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarker Development for Muscular Dystrophies

NCT05019625
View directory record Official record
Conditions
Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
465 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2028
U.S. locations
5
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 24, 2025 · Synced Oct 13, 2026, 2:41 PM 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 8, 2026 · Synced Oct 13, 2026, 2:41 PM EDT
Completed No phase listed Observational

Cardiac Magnetic Resonance in Children With Muscular Dystrophy

NCT01081080
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 11, 2013 · Synced Oct 13, 2026, 2:41 PM 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 13, 2026, 2:41 PM EDT
Completed No phase listed Observational

Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy

NCT05280730
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
No intervention
Other
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Oct 13, 2026, 2:41 PM EDT
Completed No phase listed Observational

Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy

NCT01451281
View directory record Official record
Conditions
Muscular Dystrophy, Muscular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
5 Years to 17 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 22, 2019 · Synced Oct 13, 2026, 2:41 PM EDT
Completed No phase listed Observational

Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03319030
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
2 Years to 17 Years · Male only
Enrollment
43 participants
Timeline
2017 – 2018
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 4, 2019 · Synced Oct 13, 2026, 2:41 PM EDT