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2026 data Public-data reference. official source

Duchenne Muscular Dystrophy

Open-data reference.

49 US clinical trials · 22 currently recruiting

Active & Recent Trials

RECRUITING 10,000 participants

The Duchenne Registry

The Duchenne Registry

NCT02069756

RECRUITING 5,000 participants

CureDuchenne Link®: A Resource for Research

CureDuchenne

NCT04972604

RECRUITING 1,000 participants

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

xCures

NCT06539169

RECRUITING 550 participants

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

University of Florida

NCT01484678

RECRUITING 465 participants

Biomarker Development for Muscular Dystrophies

Massachusetts General Hospital

NCT05019625

RECRUITING 250 participants

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

Catalyst Pharmaceuticals

NCT06564974

RECRUITING Phase 2 206 participants

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

Italfarma

NCT03373968

RECRUITING 200 participants

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

REGENXBIO

NCT05683379

RECRUITING 100 participants

Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

Massachusetts General Hospital

NCT05016908

RECRUITING 80 participants

Wearable Technology to Evaluate Hyperglycemia and HRV in DMD

Vanderbilt University Medical Center

NCT06124196

RECRUITING Phase 2 65 participants

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

REGENXBIO

NCT05693142

RECRUITING Phase 1 60 participants

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

Solid Biosciences

NCT06138639

RECRUITING Phase 2 51 participants

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Satellos Bioscience

NCT07287189

RECRUITING Phase 2 50 participants

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

Hoffmann-La Roche

NCT06450639

RECRUITING Phase 2 50 participants

Vasodilator and Exercise Study for DMD (VASO-REx)

University of Florida

NCT06290713

RECRUITING Phase 1 26 participants

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Wave Life Sciences

NCT04906460

RECRUITING Phase 4 26 participants

Once Weekly Infant Corticosteroid Trial for DMD

Anne M. Connolly

NCT05412394

RECRUITING Phase 1 20 participants

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NS Pharma

NCT06053814

RECRUITING Phase 2 20 participants

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

NS Pharma

NCT05996003

RECRUITING Phase 1 12 participants

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

Insmed Gene Therapy

NCT06817382

RECRUITING 10 participants

Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim

Vanderbilt University Medical Center

NCT06093100

RECRUITING Phase 1 8 participants

Trial of Cell Based Therapy for DMD

Masonic Cancer Center, University of Minnesota

NCT06692426

ACTIVE NOT RECRUITING 250 participants

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

Nationwide Children's Hospital

NCT02972580

ACTIVE NOT RECRUITING Phase 3 148 participants

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

Sarepta Therapeutics

NCT05881408

ACTIVE NOT RECRUITING Phase 3 114 participants

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Pfizer

NCT04281485

ACTIVE NOT RECRUITING Phase 2 76 participants

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

Edgewise Therapeutics

NCT05540860

ACTIVE NOT RECRUITING 75 participants

Pediatric Radio Frequency Coils Generic

Children's Hospital Medical Center, Cincinnati

NCT01633866

ACTIVE NOT RECRUITING Phase 2 43 participants

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

Edgewise Therapeutics

NCT06100887

ACTIVE NOT RECRUITING Phase 2 39 participants

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

Avidity Biosciences

NCT06244082

ACTIVE NOT RECRUITING Phase 2 25 participants

Tadalafil as Adjuvant Therapy for DMD

University of Florida

NCT05195775

ACTIVE NOT RECRUITING Phase 2 13 participants

Open-label Extension of the HOPE-2 Trial

Capricor

NCT04428476

ACTIVE NOT RECRUITING Phase 1 12 participants

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Solid Biosciences

NCT03368742

ACTIVE NOT RECRUITING Phase 4 9 participants

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

NS Pharma

NCT04687020

COMPLETED 150 participants

Duchenne Muscular Dystrophy Video Assessment Registry

The Emmes Company

NCT05712447

COMPLETED Phase 3 126 participants

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

Sarepta Therapeutics

NCT05096221

COMPLETED 73 participants

Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy

Boston Children's Hospital

NCT01491555

COMPLETED Phase 1 70 participants

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

Avidity Biosciences

NCT05670730

COMPLETED NA 53 participants

Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy

Nationwide Children's Hospital

NCT01645098

COMPLETED Phase 3 52 participants

Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

Ohio State University

NCT02354352

COMPLETED 50 participants

This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy

Washington University School of Medicine

NCT01098708

COMPLETED NA 42 participants

Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy

Subha Raman

NCT01521546

COMPLETED 39 participants

Wearable Technology to Assess Gait Function in SMA and DMD

Columbia University

NCT04193085

COMPLETED 31 participants

NSAA NON-Interventional Study Protocol

Red Nucleus Enterprise Solutions

NCT06054971

COMPLETED Phase 2 16 participants

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NS Pharma

NCT03167255

COMPLETED Phase 2 14 participants

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

PTC Therapeutics

NCT02819557

COMPLETED Phase 2 8 participants

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

Capricor

NCT06304064

COMPLETED Phase 2 6 participants

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

PTC Therapeutics

NCT03796637

COMPLETED Phase 1 4 participants

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

Sarepta Therapeutics

NCT03375164

COMPLETED Phase 1 4 participants

Gentamicin Treatment of Muscular Dystrophy

National Institute of Neurological Disorders and Stroke (NINDS)

NCT00005574

Phase Distribution

PhaseTrial count
Early Phase 1 9
Phase 2 15
Phase 3 4
Phase 4 2

Top Sponsors

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Duchenne Muscular Dystrophy Trial Landscape

ClinicalTrials.gov lists 49 US studies indexed under Duchenne Muscular Dystrophy, and 22 of those are currently open to recruitment — roughly 45% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

The phase distribution for Duchenne Muscular Dystrophy shows 6 late-stage studies (Phase 3 and Phase 4 combined) alongside 24 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

Top sponsor activity for Duchenne Muscular Dystrophy is led by NS Pharma with 4 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 49 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Duchenne Muscular Dystrophy?

PlainTrial tracks 49 US clinical trials for Duchenne Muscular Dystrophy, of which 22 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Duchenne Muscular Dystrophy?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

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