Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Jun 26, 2026, 3:35 PM EDT

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Showing 1–24 of 236 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
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
Completed Phase 2 Interventional Results available

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

NCT06304064
Conditions
Duchenne Muscular Dystrophy
Interventions
Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
2
States / cities
Gainesville, Florida • Cincinnati, Ohio
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisone
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years to 10 Years · Male only
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Muscular Dystrophy, Duchenne, Ambulatory Care
Interventions
Placebo, Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
10 Years to 18 Years · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Gainesville, Florida + 6 more
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
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
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-065/NCNP-01
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 10 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Sacramento, California • Chicago, Illinois • St Louis, Missouri + 2 more
Conditions
Becker Muscular Dystrophy
Interventions
(-)-epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Sacramento, California
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
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
2 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rockville, Maryland
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
Conditions
Duchenne Muscular Dystrophy
Interventions
RGX-202
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Year and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
13
States / cities
Little Rock, Arkansas • Palo Alto, California • Aurora, Colorado + 10 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
7 Years to 22 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
St Louis, Missouri
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
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Limb-Girdle Muscular Dystrophy

NCT00893334
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
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
Conditions
Duchenne Muscular Dystrophy, Exon 44
Interventions
AOC 1044, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 55 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Palo Alto, California + 7 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Ketamine, Dexmedetomidine
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Columbus, Ohio
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
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
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
Conditions
Stress
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
228 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
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
1
States / cities
Rochester, New York