Duchenne Clinical Trials

1 recruiting

Frequently Asked Questions

Common questions about Duchenne clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 56 trials

Recruiting
Phase 2

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Duchenne Muscular DystrophyDMDNeuromuscular Diseases+2 more
Satellos Bioscience, Inc.51 enrolled19 locationsNCT07287189
Recruiting
Phase 2

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

Duchenne Muscular Dystrophy
Hoffmann-La Roche50 enrolled25 locationsNCT06450639
Recruiting
Phase 1

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy
Insmed Gene Therapy LLC12 enrolled9 locationsNCT06817382
Recruiting
Phase 1Phase 2

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy
BioMarin Pharmaceutical18 enrolled8 locationsNCT06280209
Recruiting
Phase 1Phase 2

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

Duchenne Muscular Dystrophy (DMD)
Entrada Therapeutics, Inc.24 enrolled14 locationsNCT07037862
Recruiting
Phase 1Phase 2

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

Duchenne Muscular Dystrophy (DMD)
Entrada Therapeutics, Inc.24 enrolled15 locationsNCT07038824
Recruiting
Phase 2

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular DystrophyDMDExon 44
NS Pharma, Inc.20 enrolled25 locationsNCT05996003
Recruiting
Phase 3

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

Duchenne Muscular Dystrophy
Solid Biosciences Inc.80 enrolled2 locationsNCT07160634
Recruiting
Phase 1Phase 2

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Duchenne Muscular Dystrophy
Solid Biosciences Inc.60 enrolled15 locationsNCT06138639
Recruiting
Phase 1

Trial of Cell Based Therapy for DMD

Duchenne Muscular Dystrophy
Masonic Cancer Center, University of Minnesota8 enrolled1 locationNCT06692426
Recruiting
Phase 1

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

Muscular Dystrophy, Duchenne
Sarepta Therapeutics, Inc.83 enrolled5 locationsNCT04626674
Recruiting

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Duchenne Muscular Dystrophy
Catalyst Pharmaceuticals, Inc.250 enrolled27 locationsNCT06564974
Recruiting
Phase 1

A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants

Duchenne Muscular Dystrophy (DMD)
Grünenthal GmbH24 enrolled1 locationNCT07347548
Recruiting

Natural History of Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy
Genethon220 enrolled15 locationsNCT03882827
Recruiting
Not Applicable

Urinary Titin Biomarker in DMD

Duchenne Muscular Dystrophy (DMD)Becker's Muscular Dystrophy (BMD)
Children's Hospital of Philadelphia50 enrolled1 locationNCT07332013
Recruiting

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

Duchenne Muscular Dystrophy (DMD)Spinal Muscular Atrophy Type 3
Columbia University106 enrolled3 locationsNCT06839469
Recruiting
Not Applicable

Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy

Duchenne Muscular DystrophyOccupational TherapyGamification
Başak Çağla Arslan20 enrolled1 locationNCT06402942
Recruiting
Phase 2Phase 3

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Duchenne Muscular Dystrophy
Italfarmaco206 enrolled39 locationsNCT03373968
Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting
Not Applicable

Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening

Duchenne Muscular Dystrophy (DMD)Spinal Muscular Atrophy (SMA)
Centre Hospitalier Universitaire de Liege100 enrolled1 locationNCT07286565