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ClinicalTrials.gov public records Last synced Oct 6, 2026, 10:16 AM EDT

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Showing 49–72 of 237 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Duchenne Muscular Dystrophy (DMD) Clear all
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 6, 2026, 10:16 AM EDT
Recruiting No phase listed Observational

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

NCT07092540
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
0 Days to 3 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Rochester, New York • Stony Brook, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 6, 2026, 10:16 AM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 29, 2020 · Synced Oct 6, 2026, 10:16 AM EDT
Completed Phase 2 Interventional Results available

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

NCT02167217
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisolone
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Month to 30 Months · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
6
States / cities
Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2018 · Synced Oct 6, 2026, 10:16 AM EDT
Terminated Phase 3 Interventional

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

NCT02814019
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Idebenone 150 mg film-coated tablets, placebo
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
10 Years and older · Male only
Enrollment
255 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
20
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2021 · Synced Oct 6, 2026, 10:16 AM EDT
Terminated Phase 3 Interventional Results available

CoQ10 and Prednisone in Non-Ambulatory DMD

NCT00308113
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisone, Coenzyme Q10
Drug · Dietary Supplement
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
10 Years to 18 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 8, 2013 · Synced Oct 6, 2026, 10:16 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren, PLACEBO
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
360 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
22
States / cities
Phoenix, Arizona • Los Angeles, California • Oakland, California + 19 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 6, 2026, 10:16 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Limb-Girdle Muscular Dystrophy

NCT00893334
View directory record Official record
Conditions
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 6, 2026, 10:16 AM EDT
Completed Phase 1Phase 2 Interventional Results available

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

NCT03375164
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
3 Months to 7 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 6, 2026, 10:16 AM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

NCT04060199
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 6, 2026, 10:16 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, 10:16 AM 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 6, 2026, 10:16 AM EDT
Completed Phase 1 Interventional

Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

NCT00428935
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAV2.5-CMV-minidystrophin (d3990)
Biological
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
5 Years to 15 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 5, 2013 · Synced Oct 6, 2026, 10:16 AM EDT
Recruiting No phase listed Observational

Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

NCT05016908
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
100 participants
Timeline
2019 – 2027
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 24, 2025 · Synced Oct 6, 2026, 10:16 AM 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 12, 2019 · Synced Oct 6, 2026, 10:16 AM EDT
Active, not recruiting No phase listed Observational

Defining Endpoints in Becker Muscular Dystrophy

NCT05257473
View directory record Official record
Conditions
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years and older · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
9
States / cities
Orange, California • Aurora, Colorado • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 6, 2026, 10:16 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Pediatric Radio Frequency Coils Generic

NCT01633866
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2031
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 6, 2026, 10:16 AM EDT
Recruiting Phase 1 Interventional

Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

NCT07172971
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
SGLT-2 inhibitor, SGLT2 inhibitor
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
8 Years to 18 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 6, 2026, 10:16 AM EDT
Completed Phase 1 Interventional

Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons

NCT00451074
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Gentamicin infusions twice a week for six months
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
5 Years to 20 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Scottsdale, Arizona • Kansas City, Kansas • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 23, 2012 · Synced Oct 6, 2026, 10:16 AM EDT
Recruiting No phase listed Observational

Duchenne Electronic Health Record Study

NCT07609394
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
Interventions
Observational study with patients who may be treated with various disease-modifying therapies
Other
Lead sponsor
The Duchenne Registry
Other
Eligibility
Not listed
Enrollment
2,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2072
U.S. locations
10
States / cities
Little Rock, Arkansas • Sacramento, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 6, 2026, 10:16 AM EDT
Approved For Marketing No phase listed Expanded access

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

NCT02592941
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older
U.S. locations
42
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 36 more
Source: ClinicalTrials.gov public record
Updated Feb 28, 2018 · Synced Oct 6, 2026, 10:16 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 9, 2013 · Synced Oct 6, 2026, 10:16 AM EDT
Completed Phase 1 Interventional

Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis

NCT01519349
View directory record Official record
Conditions
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Interventions
rAAV1.CMV.huFollistatin344
Biological
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 2, 2023 · Synced Oct 6, 2026, 10:16 AM 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 6, 2026, 10:16 AM EDT