Duchenne muscular dystrophy trials in United States

In United States 36 Also abroad 87

36 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

No phaseEnrolling by invitation
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Where
Baltimore, Charlottesville, Cincinnati, Columbus, Durham, Fort Worth, Gainesville, Hershey, Houston, Iowa City, Kansas City, Little Rock, Los Angeles, New York, Orange, Philadelphia, Pittsburgh, Sacramento, Seattle, St Louis, The Bronx, Washington D.C., Winston-Salem
People
300
Run by
Sarepta Therapeutics, Inc.
See details and how to joinNCT06606340
No phaseTaking people
Duchenne Electronic Health Record Study
Where
Aurora, Dallas, Durham, Iowa City, Little Rock, New Haven, Sacramento, Salt Lake City, Seattle, Washington D.C.
People
2,500
Run by
The Duchenne Registry
See details and how to joinNCT07609394
No phaseTaking people
Biomarker Development for Muscular Dystrophies
Where
Boston, Pittsburgh, Winston-Salem
Age
5 years and over
People
465
Run by
Massachusetts General Hospital
See details and how to joinNCT05019625
No phaseEnrolling by invitation
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
Where
Akron, Albuquerque, Ann Arbor, Aurora, Birmingham, Boston, Charlottesville, Chicago, Durham, Fort Worth, Hartford, Hershey, Indianapolis, Kansas City, Little Rock, Los Angeles, Louisville, Madison, Miami, Milwaukee, Nashville, Norfolk, Orlando, Philadelphia, Portland, Seattle, Stony Brook, St. Petersburg, Washington D.C.
Age
4 years and over
People
500
Run by
Sarepta Therapeutics, Inc.
See details and how to joinNCT06270719
Phase 1/2Taking people
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Where
Little Rock, St Louis
Age
2 years to 7 years
People
18
Run by
Precision BioSciences, Inc.
See details and how to joinNCT07429240
Phase 2Enrolling by invitation
An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101
Where
Columbus, Los Angeles, Memphis, St Louis
Age
16 years to 40 years
People
30 worldwide
Run by
Satellos Bioscience, Inc.
See details and how to joinNCT06867107
Phase 2Taking people
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
Where
Gainesville
Age
6 years and over
People
20
Run by
University of Florida
See details and how to joinNCT07766980
Phase 4Taking people
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
Where
Houston
Age
4 years and over
People
20
Run by
Sarepta Therapeutics, Inc.
See details and how to joinNCT07542314
Phase 3Taking people
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Where
Flower Mound, Little Rock, Norfolk
Age
7 years to 11 years
People
80 worldwide
Run by
Solid Biosciences Inc.
See details and how to joinNCT07160634 · EU 2025-522949-22-00
No phaseTaking people
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Where
Charlotte, Charlottesville, Chicago, Cincinnati, Dallas, Denton, Durham, Gainesville, Grand Rapids, Hershey, Indianapolis, Kansas City, Little Rock, Loma Linda, Los Angeles, Madera, Miami, Milwaukee, Minneapolis, North Worcester, Orlando, Palo Alto, Philadelphia, Phoenix, Sacramento, San Antonio, Seattle, St. Petersburg, The Bronx, Washington D.C.
Age
2 years and over
People
250 worldwide
Run by
Catalyst Pharmaceuticals, Inc.
See details and how to joinNCT06564974
Phase 2Enrolling by invitation
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
Where
Atlanta, Aurora, Austin, Baltimore, Chicago, Cincinnati, Columbus, Denton, Gainesville, Hillsborough, Indianapolis, Iowa City, Kansas City, La Jolla, Little Rock, Los Angeles, Orange, Palo Alto, Philadelphia, Richmond, Rochester, Sacramento, St Louis, Worcester
People
260 worldwide
Run by
Edgewise Therapeutics, Inc.
See details and how to joinNCT06066580
Phase 1Taking people
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Where
Chicago, Columbus, Durham, Flower Mound, Gainesville, La Jolla, Little Rock, Los Angeles, Norfolk, Palo Alto, Sacramento, St Louis
Age
2 years and over
People
83
Run by
Sarepta Therapeutics, Inc.
See details and how to joinNCT04626674
Phase 1Taking people
Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy
Where
Nashville
Age
8 years to 18 years
People
10
Run by
Vanderbilt University Medical Center
See details and how to joinNCT07172971
No phaseTaking people
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
Where
Cincinnati, Columbus, Rochester, Stony Brook
Age
0 days to 3 years
People
105
Run by
University of Rochester
See details and how to joinNCT07092540
Phase 4Taking people
Once Weekly Infant Corticosteroid Trial for DMD
Where
Chicago, Cincinnati, Columbus, Dallas
Age
1 month to 30 months
People
26
Run by
Anne M. Connolly
See details and how to joinNCT05412394
No phaseTaking people
Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials
Where
Chicago, Columbus, Durham, Indianapolis, Nashville, Richmond, Sacramento, Seattle, Washington D.C.
People
1,000
Run by
Vanderbilt University Medical Center
See details and how to joinNCT07674758
No phaseEnrolling by invitation
Parent and Infant Inter(X)Action Intervention (PIXI)
Where
Research Triangle Park
Age
Up to 99 years
People
120
Run by
RTI International
See details and how to joinNCT03836300
Phase 1/2Taking people
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Where
Atlanta, Chicago, Columbus, Little Rock, Los Angeles, Norfolk, Philadelphia, Portland, Sacramento, San Diego, Seattle, St Louis
Age
0 years to 17 years
People
60 worldwide
Run by
Solid Biosciences Inc.
See details and how to joinNCT06138639 · EU 2024-514501-57-00
No phaseTaking people
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Where
Boston, Chicago, Durham, Little Rock, New York, Portland, Seattle, Washington D.C., Worcester
Age
6 years and over
People
300
Run by
ITF Therapeutics LLC
See details and how to joinNCT07127978
Phase 1/2Not yet taking people in United States
Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy
Where
Nashville
Age
18 years and over
People
30
Run by
Vanderbilt University Medical Center
See details and how to joinNCT07642635
Phase 3Taking people
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Where
Hillsborough
Age
4 years to 18 years
People
90
Run by
Dyne Therapeutics
See details and how to joinNCT07608432 · EU 2025-524096-23-00
No phaseTaking people
Wearable Technology to Evaluate Hyperglycemia and HRV in DMD
Where
Nashville
Age
10 years and over
People
80
Run by
Vanderbilt University Medical Center
See details and how to joinNCT06124196
Phase 2Taking people
Vasodilator and Exercise Study for DMD (VASO-REx)
Where
Gainesville
Age
6 years and over
People
50
Run by
University of Florida
See details and how to joinNCT06290713
No phaseTaking people
The Duchenne Registry
Where
Washington D.C.
People
10,000
Run by
The Duchenne Registry
See details and how to joinNCT02069756
No phaseTaking people
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
Where
Boston, New York, Palo Alto
Age
5 years and over
People
106
Run by
Columbia University
See details and how to joinNCT06839469