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Duchenne Muscular Dystrophy: early-phase trial registry
22 of 49 indexed Duchenne Muscular Dystrophy trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #281 of 28,707 conditions by registered trial count.
49 US clinical trials · 22 currently recruiting · #281 of 28,707 conditions by registered trial count
Duchenne Muscular Dystrophy: 49 US clinical trials tracked, 22 recruiting.
Duchenne Muscular Dystrophy is the subject of 49 registered US clinical trials on ClinicalTrials.gov, 22 of them currently open to new participants. 6 are in Phase 3-4 (later-stage) and 24 in Phase 1-2 (earlier-stage). The most active sponsor is NS Pharma, running 4 of these trials.
Enrollment posture brief
Registry condition duchenne-muscular-dystrophy
OPEN 22 | RECR 45% | LATE 6 | EARLY 24 | RANK #281 | SPON 4
Volume-matched condition peers
Nearest open-share peer: Psoriatic Arthritis (45% recruiting · 49 trials)
Duchenne Muscular Dystrophy lists 22 open slots (45% of 49 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Duchenne Muscular Dystrophy on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Duchenne Muscular Dystrophy is recruiting above its size-band peers.
22 of 49 indexed trials (44.9%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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Sponsorship of Duchenne Muscular Dystrophy trials is spread across many organizations.
NS Pharma accounts for 4 of 49 trials (8.2%). 33 distinct sponsors appear on at least one study for this condition label.
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Most Duchenne Muscular Dystrophy trials plan to enrol fewer than 100 people.
Median target enrolment is 50 among the 49 trials that report a genuine target (sentinel values excluded). 35 of those (71.4%) plan for fewer than 100 participants.
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Duchenne Muscular Dystrophy splits between interventional and observational designs.
32 interventional and 17 observational studies make up this index (65.3% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
49 total, page 1 of 1
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 |
|---|---|
| Phase 1 | 9 |
| Phase 2 | 15 |
| Phase 3 | 4 |
| Phase 4 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Duchenne Muscular Dystrophy records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Duchenne Muscular Dystrophy trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Duchenne Muscular Dystrophy page
49 registered trials, 22 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 22 Duchenne Muscular Dystrophy trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- NS Pharma sponsors the most Duchenne Muscular Dystrophy trials on record, review their full trial history before evaluating a specific study. See NS Pharma's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Duchenne Muscular Dystrophy condition card totals 49 registered trials; most-linked intervention Placebo; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.