Muscular Dystrophy Clinical Trials

10 recruiting

Muscular Dystrophy Trials at a Glance

94 actively recruiting trials for muscular dystrophy are listed on ClinicalTrialsFinder across 6 cities in 31 countries. The largest study group is Not Applicable with 17 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 Boston Children's Hospital.

Browse muscular dystrophy trials by phase

Treatments under study

About Muscular Dystrophy Clinical Trials

Looking for clinical trials for Muscular Dystrophy? There are currently 10 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 94 trials

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 enrolled1 locationNCT07429240
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

Investigational Use of Neuromuscular Ultrasound

Peripheral NeuropathyNormal PhysiologyMuscular Dystrophy+1 more
National Institute of Neurological Disorders and Stroke (NINDS)150 enrolled1 locationNCT05237973
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

Amino Acids and Exercise in FSHD

Muscular DystrophyFSH Muscular Dystrophy
University of Pavia48 enrolled1 locationNCT07543016
Recruiting

Invasive Home Ventilation in Denmark

Spinal Cord Injuries (SCI)Neuromuscular Diseases (NMD)MSA - Multiple System Atrophy+5 more
Rigshospitalet, Denmark450 enrolled1 locationNCT07467187
Recruiting

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

Duchene Muscular Dystrophy
ITF Therapeutics LLC300 enrolled7 locationsNCT07127978
Recruiting

Study of Inherited Neurological Disorders

Motor Neuron DiseaseMuscular DystrophyMuscular Disease
National Institute of Neurological Disorders and Stroke (NINDS)3,500 enrolled2 locationsNCT00004568
Recruiting
Phase 2

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Duchenne Muscular DystrophyDMDNeuromuscular Diseases+2 more
Satellos Bioscience, Inc.51 enrolled25 locationsNCT07287189
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
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

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
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 enrolled7 locationsNCT04626674
Recruiting

DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies

Duchenne Muscular Dystrophy (DMD)
Aristotle University Of Thessaloniki65 enrolled1 locationNCT07515235
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

Molecular and Genetic Studies of Congenital Myopathies

Nemaline MyopathyCentronuclear MyopathyCentral Core Disease+5 more
Boston Children's Hospital4,000 enrolled1 locationNCT00272883
Recruiting
Not Applicable

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

Myasthenia GravisAmyotrophic Lateral SclerosisNeuromuscular Diseases (NMD)+15 more
University of Missouri-Columbia50 enrolled1 locationNCT07478172
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