Muscular Dystrophy Clinical Trials

112 recruitingLast updated: July 11, 2026

There are 112 actively recruiting muscular dystrophy clinical trials across 34 countries. Studies span Not Applicable, Phase 1, Phase 2, Phase 3, Early Phase 1, Phase 4. Top locations include Chicago, Illinois, United States, Little Rock, Arkansas, United States, Atlanta, Georgia, United States. Updated daily from ClinicalTrials.gov.


Muscular Dystrophy Trials at a Glance

112 actively recruiting trials for muscular dystrophy are listed on ClinicalTrialsFinder across 6 cities in 34 countries. The largest study group is Not Applicable with 21 trials, with the heaviest enrollment activity in Chicago, Little Rock, and Atlanta. Lead sponsors running muscular dystrophy studies include Italfarmaco, Radboud University Medical Center, and Vanderbilt University Medical Center.

Browse muscular dystrophy trials by phase

Treatments under study

About Muscular Dystrophy Clinical Trials

Looking for clinical trials for Muscular Dystrophy? There are currently 12 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Muscular Dystrophy trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Muscular Dystrophy 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 112 trials

Recruiting

Study of Inherited Neurological Disorders

Peripheral nervous system diseaseMotor Neuron DiseaseMuscular Dystrophy+1 more
National Institute of Neurological Disorders and Stroke (NINDS)3,500 enrolled2 locationsNCT00004568
Recruiting

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

Duchene Muscular Dystrophy
ITF Therapeutics LLC300 enrolled9 locationsNCT07127978
Recruiting
Phase 1

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

Duchenne Muscular Dystrophy (DMD)
Larry W. Markham10 enrolled1 locationNCT07172971
Recruiting

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

Duchenne Muscular Dystrophy
REGENXBIO Inc.200 enrolled1 locationNCT05683379
Recruiting
Phase 2Phase 3

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy
REGENXBIO Inc.65 enrolled17 locationsNCT05693142
Recruiting

Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials

Becker muscular dystrophyCardiomyopathyDuchenne Muscular Dystrophy (DMD)+1 more
Vanderbilt University Medical Center1,000 enrolled9 locationsNCT07674758
Recruiting

Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies

Becker muscular dystrophyDuchene Muscular Dystrophy
Nantes University Hospital60 enrolled1 locationNCT07378553
Recruiting
Phase 1Phase 2

A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy
AO GENERIUM32 enrolled6 locationsNCT07673809
Recruiting
Phase 1Phase 2

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)

Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Precision BioSciences, Inc.18 enrolled2 locationsNCT07429240
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) - Non-Ambulatory Cohort

Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc.83 enrolled7 locationsNCT04626674
Recruiting
Not Applicable

Personalized Training for People With Rare Neuromuscular Disorders

facioscapulohumeral muscular dystrophyNeuromuscular Diseases (NMD)Charcot Marie Tooth Disease (CMT)+1 more
Oslo University Hospital120 enrolled5 locationsNCT06708468
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 2

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Duchenne Muscular DystrophyDMDNeuromuscular Diseases+2 more
Satellos Bioscience, Inc.51 enrolled21 locationsNCT07287189
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 enrolled10 locationsNCT06817382
Recruiting

A Remote Study Using Technology to Assess Outcomes in DMD

Duchenne Muscular Dystrophy (DMD)
University of Oxford60 enrolled1 locationNCT07423026
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 enrolled5 locationsNCT07160634
Recruiting
Not Applicable

BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

Amyotrophic Lateral SclerosisSpinal Cord Injurieslocked-in Syndrome+3 more
Leigh R. Hochberg, MD, PhD.27 enrolled6 locationsNCT00912041
Recruiting
Phase 3

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

facioscapulohumeral muscular dystrophyFascioscapulohumeral Muscular DystrophyFacio-Scapulo-Humeral Dystrophy+14 more
Avidity Biosciences, Inc.200 enrolled46 locationsNCT07038200
Recruiting

Duchenne Electronic Health Record Study

Becker muscular dystrophyDuchenne Muscular Dystrophy (DMD)Dystrophinopathy+1 more
The Duchenne Registry2,500 enrolled10 locationsNCT07609394
Recruiting
Phase 3

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

Muscular Dystrophy, DuchenneDMDNeuromuscular Diseases (NMD)+10 more
Dyne Therapeutics90 enrolled1 locationNCT07608432