Muscular Dystrophy, Duchenne clinical trials
The registry lists 481 studies for Muscular Dystrophy, Duchenne across more than 10 countries. 107 are recruiting or about to start. 448 of them are mainly about Muscular Dystrophy, Duchenne (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Status
Who runs them
Sarepta Therapeutics, Inc. (27) · Hacettepe University (20) · PTC Therapeutics (19) · Cooperative International Neuromuscular Research Group (15) · BioMarin Pharmaceutical (10) · University of Florida (9) · NS Pharma, Inc. (9) · Santhera Pharmaceuticals (9)
What is being tested here
Drugs under test in Muscular Dystrophy, Duchenne studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Givinostat | 3 | 3 | 7 |
| Tadalafil | 1 | 2 | 7 |
| Sevasemten | 1 | 0 | 4 |
| SAT-3247 | 1 | 0 | 3 |
| Delpacibart zotadirsen | 1 | 1 | 3 |
| Tegacorat | 1 | 0 | 3 |
| EN001 | 1 | 0 | 2 |
| Prednisolone | 1 | 0 | 2 |
| Semaglutide | 1 | 0 | 1 |
| Tacrolimus | 1 | 0 | 1 |
Drug classes tested here HDAC inhibitors 3 open · PDE5 inhibitors 1 open · Corticosteroids 1 open · Beta blockers 1 open · Mineralocorticoid receptor antagonists (MRAs) 3
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The 10 nearest, phase 3 first
- May 2027A 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) · Phase 3 · Sarepta Therapeutics, Inc.
- Nov 2026A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy · Peking Union Medical College Hospital
- Nov 2026Vasodilator and Exercise Study for DMD (VASO-REx) · University of Florida
- Nov 2026A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) · Hoffmann-La Roche
- Nov 2026Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy · Aga Khan University
- Nov 2026Microdystrophin Gene Transfer Study in Adolescents and Children With DMD · Solid Biosciences Inc.
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Nov 2026Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy? · Antalya Training and Research Hospital
- Dec 2026Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy · Massachusetts General Hospital
- Dec 2026Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients? · taVNS AB
Who these studies take
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger. 60 (91%) take children under 18. 1 (2%) are for women only and 43 (65%) for men only; 12 (18%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 18 (27%) | 47 (71%) |
| 18 | 21 (32%) | 46 (70%) |
| 30 | 20 (30%) | 37 (56%) |
| 40 | 19 (29%) | 35 (53%) |
| 50 | 19 (29%) | 34 (52%) |
| 65 | 19 (29%) | 32 (48%) |
| 75 | 18 (27%) | 31 (47%) |
| 85 | 17 (26%) | 30 (45%) |
Across all 481 studies, recruiting or not: 66% stop at 75 or younger and 65% at 65; 90% take children; 1% are for women only and 76% for men only; 16% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
How long Muscular Dystrophy, Duchenne studies take
Of 47 phase 2 interventional studies under Muscular Dystrophy, Duchenne, half reached primary completion within 31 months of starting, counting those still running.
| Start to primary completion | Every sponsor | Industry | Non-industry |
|---|---|---|---|
| Phase 1 | 19 mo11–35 · 28 studies | — | — |
| Phase 1/2 | 50 mo24–63 · 23 studies | — | — |
| Phase 2 | 31 mo20–42 · 47 studies | 31 mo20–42 · 40 studies | — |
| Phase 3 | 54 mo38–73 · 26 studies | — | — |
| Interventional, no phase | 20 mo10–44 · 47 studies | — | 19 mo10–44 · 45 studies |
| Observational | 40 mo12–120 · 63 studies | — | 37 mo11–120 · 52 studies |
Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out
What changed recently
From the registry's own updates since Oct 13, 2026.
Started recruiting1
Results posted2
- A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.Nov 12, 2026NCT05689164
- Tadalafil as Adjuvant Therapy for DMDOct 27, 2026NCT05195775
Newly registered4
- A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular DystrophyNov 3, 2026NCT07911995
- Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?Oct 23, 2026NCT07892196
- Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.Oct 22, 2026NCT07890883
- Respiratory Progression in Adults With Duchenne Muscular Dystrophy.Oct 19, 2026NCT07879989
Studies
Recruiting first, then most recently updated. Showing 50 of 481. Browse all 481 → or search them.
- Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular DystrophyRecruitingNCT07642635Phase 1/21 country
- Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing SettingRecruitingNCT07542314Phase 41 country
- A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 SkippingRecruitingNCT07587242Phase 310 countries
- Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) PatientsRecruitingNCT07188012Early Phase 11 country
- Safety and Efficacy of KER-065 in Participants With Duchenne Muscular DystrophyRecruitingNCT07704099Phase 22 countries
- Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle FunctionNot yet recruitingNCT077400571 country
- A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular DystrophyNot yet recruitingNCT07911995Phase 2
- Duchenne Electronic Health Record StudyRecruitingNCT076093941 country
- A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical PracticeEnrolling by invitationNCT066063401 country
- Biomarker Development for Muscular DystrophiesRecruitingNCT050196251 country
- Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle DiseasesRecruitingNCT06833489Not applicable1 country
- An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular DystrophyEnrolling by invitationNCT062707191 country
- Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?RecruitingNCT078921961 country
- Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.RecruitingNCT078908831 country
- PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)RecruitingNCT07429240Phase 1/21 country
- Development of Non-Invasive Prenatal Diagnosis for Single Gene DisordersRecruitingNCT061474141 country
- Respiratory Progression in Adults With Duchenne Muscular Dystrophy.RecruitingNCT078799891 country
- An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101Enrolling by invitationNCT06867107Phase 22 countries
- Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 SkippingAvailableNCT072507371 country
- Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy PatientsRecruitingNCT07766980Phase 21 country
- Registry for Duchenne and Becker Muscular DystrophyRecruitingNCT074021222 countries
- A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)RecruitingNCT07160634Phase 33 countries
- Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMITRecruitingNCT065649742 countries
- Open-Label Extension of EDG-5506 in Participants With Becker Muscular DystrophyEnrolling by invitationNCT06066580Phase 212 countries
- Urotherapy Education for Families of Children With Duchenne Muscular DystrophyNot yet recruitingNCT07821125Not applicable1 country
- 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 CohortRecruitingNCT04626674Phase 11 country
- Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular DystrophyNot yet recruitingNCT07787689Not applicable1 country
- Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne CardiomyopathyRecruitingNCT07172971Phase 11 country
- The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular DystrophyRecruitingNCT070925401 country
- Once Weekly Infant Corticosteroid Trial for DMDRecruitingNCT05412394Phase 41 country
- A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45RecruitingNCT07038824Phase 1/25 countries
- A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44RecruitingNCT07037862Phase 1/24 countries
- Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug TrialsRecruitingNCT076747581 country
- Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular DystrophyRecruitingNCT05933057Phase 312 countries
- An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular DystrophyEnrolling by invitationNCT07573631Phase 25 countries
- Parent and Infant Inter(X)Action Intervention (PIXI)Enrolling by invitationNCT03836300Not applicable1 country
- A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)RecruitingNCT06138639Phase 1/24 countries
- A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular DystrophyRecruitingNCT071279781 country
- Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)Not yet recruitingNCT07682129Phase 25 countries
- A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular DystrophyRecruitingNCT07673809Phase 1/22 countries
- Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular DystrophiesRecruitingNCT073785531 country
- Digital Monitoring of Upper Limb Function in Non-Ambulant DMDNot yet recruitingNCT07664124Not applicable1 country
- A Remote Study Using Technology to Assess Outcomes in DMDRecruitingNCT074230261 country
- Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)RecruitingNCT07608432Phase 31 country
- Wearable Technology to Evaluate Hyperglycemia and HRV in DMDRecruitingNCT061241961 country
- The Effect of Virtual Reality Applications on Upper Extremity Functions in Patients With Duchenne Muscular DystrophyRecruitingNCT07039799Not applicable1 country
- Vasodilator and Exercise Study for DMD (VASO-REx)RecruitingNCT06290713Phase 21 country
- The Duchenne RegistryRecruitingNCT020697561 country
- Usability and User Experience of a Virtual Reality Rehabilitation Game Platform in Individuals With Duchenne Muscular DystrophyEnrolling by invitationNCT075652721 country
- Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients?Enrolling by invitationNCT07543302Not applicable1 country