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Duchenne Muscular Dystrophy
Open-data reference.
49 US clinical trials · 22 currently recruiting
Active & Recent Trials
The Duchenne Registry
The Duchenne Registry
NCT02069756
CureDuchenne Link®: A Resource for Research
CureDuchenne
NCT04972604
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
xCures
NCT06539169
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
University of Florida
NCT01484678
Biomarker Development for Muscular Dystrophies
Massachusetts General Hospital
NCT05019625
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Catalyst Pharmaceuticals
NCT06564974
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Italfarma
NCT03373968
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
REGENXBIO
NCT05683379
Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
Massachusetts General Hospital
NCT05016908
Wearable Technology to Evaluate Hyperglycemia and HRV in DMD
Vanderbilt University Medical Center
NCT06124196
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
REGENXBIO
NCT05693142
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Solid Biosciences
NCT06138639
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Satellos Bioscience
NCT07287189
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
Hoffmann-La Roche
NCT06450639
Vasodilator and Exercise Study for DMD (VASO-REx)
University of Florida
NCT06290713
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Wave Life Sciences
NCT04906460
Once Weekly Infant Corticosteroid Trial for DMD
Anne M. Connolly
NCT05412394
NS-050/NCNP-03 in Boys With DMD (Meteor50)
NS Pharma
NCT06053814
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
NS Pharma
NCT05996003
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
Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim
Vanderbilt University Medical Center
NCT06093100
Trial of Cell Based Therapy for DMD
Masonic Cancer Center, University of Minnesota
NCT06692426
Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
Nationwide Children's Hospital
NCT02972580
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
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Pfizer
NCT04281485
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
Edgewise Therapeutics
NCT05540860
Pediatric Radio Frequency Coils Generic
Children's Hospital Medical Center, Cincinnati
NCT01633866
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
Edgewise Therapeutics
NCT06100887
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
Avidity Biosciences
NCT06244082
Tadalafil as Adjuvant Therapy for DMD
University of Florida
NCT05195775
Open-label Extension of the HOPE-2 Trial
Capricor
NCT04428476
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Solid Biosciences
NCT03368742
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
NS Pharma
NCT04687020
Duchenne Muscular Dystrophy Video Assessment Registry
The Emmes Company
NCT05712447
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
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Boston Children's Hospital
NCT01491555
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
Avidity Biosciences
NCT05670730
Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy
Nationwide Children's Hospital
NCT01645098
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
Ohio State University
NCT02354352
This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy
Washington University School of Medicine
NCT01098708
Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
Subha Raman
NCT01521546
Wearable Technology to Assess Gait Function in SMA and DMD
Columbia University
NCT04193085
NSAA NON-Interventional Study Protocol
Red Nucleus Enterprise Solutions
NCT06054971
Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
NS Pharma
NCT03167255
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
PTC Therapeutics
NCT02819557
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
Capricor
NCT06304064
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
PTC Therapeutics
NCT03796637
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Sarepta Therapeutics
NCT03375164
Gentamicin Treatment of Muscular Dystrophy
National Institute of Neurological Disorders and Stroke (NINDS)
NCT00005574
Phase Distribution
| Phase | Trial 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
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.
Read our methodology — how this data is sourced, computed, and verified.
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.