Data updated: Jun 28, 2026
Duchenne Muscular Dystrophy Clinical Trial Landscape
Duchenne Muscular Dystrophy is being studied across 163 clinical trials registered since 2008, with 49 programs currently active. The competitive pipeline includes 11 active Phase 3 trials, 27 active Phase 2 trials, and 7 active Phase 1 trials.
Top industry sponsors include Sarepta Therapeutics, Inc., NS Pharma, Inc., Italfarmaco.
Trial activity
49 active / 163 total since 2008
Active by phase 11 Ph3 / 36 27 Ph2 / 91 7 Ph1 / 29 4 Ph4 / 7
Sponsor activity
Who is running trials now — green active, blue completed, red failed/terminated.
Sorted by active Active Done Failed
Sarepta Therapeutics, Inc. 4 11 5
NS Pharma, Inc. 3 4 0
Italfarmaco 3 2 0
Solid Biosciences Inc. 3 0 0
BioMarin Pharmaceutical 2 1 4
Roche 2 1 1
Wave Life Sciences Ltd. 2 1 1
Avidity Biosciences, Inc. 2 1 0
Satellos Bioscience, Inc. 2 0 0
Entrada Therapeutics, Inc. 2 0 0
Edgewise Therapeutics, Inc. 2 0 0
Dyne Therapeutics 2 0 0
Santhera Pharmaceuticals 1 2 2
Pfizer 1 0 4
Capricor Inc. 1 2 0
How the field has grown
New-trial starts peaked in 2016 (15 registered); 2025 saw 13. The right-hand chart shows median Phase 3 enrollment by start year — the number in parentheses is that year's Phase 3 trial count (25 in total), so single-trial years (and years with no Phase 3 starts) are obvious. Both are by trial start date; the current year is partial.
New trials started by year
2016 15
2017 8
2018 11
2019 6
2020 12
2021 10
2022 5
2023 6
2024 15
2025 13
2026 8
TheraRadar.com
Median Phase 3 enrollment by start year
2016 (2) 242
2017 (3) 179
2018 (3) 161
2019 (2) 68
2020 (4) 101
2021 (3) 73
2022 (0) 0
2023 (4) 107
2024 (1) 138
2025 (1) 80
2026 (2) 80
TheraRadar.com
Full trial pipeline
Every active and completed trial across Phase 1–4, with enrollment analytics. Sortable, filterable, exportable with Pro.
NCT07160634 RECRUITING
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Solid Biosciences Inc. n=80
NCT04281485 ACTIVE NOT RECRUITING
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Pfizer n=114
NCT05967351 ENROLLING BY INVITATION
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Sarepta Therapeutics, Inc. n=400
NCT03373968 RECRUITING
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Italfarmaco n=206
NCT05693142 RECRUITING
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
REGENXBIO Inc. n=65
NCT04587908 ACTIVE NOT RECRUITING
A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
Taiho Pharmaceutical Co., Ltd. n=104
NCT05881408 ACTIVE NOT RECRUITING
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, Inc. n=148
NCT05933057 RECRUITING
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Italfarmaco n=138
NCT07587242 NOT YET RECRUITING
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 Skipping
Avidity Biosciences, Inc. n=70
NCT07608432 RECRUITING
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Dyne Therapeutics n=90
NCT01247207 COMPLETED
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
PTC Therapeutics n=270
NCT01648634 COMPLETED
Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
Assistance Publique - Hôpitaux de Paris n=51
NCT02500381 COMPLETED
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
Sarepta Therapeutics, Inc. n=228
NCT05689164 TERMINATED
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
Pfizer n=7
NCT05096221 COMPLETED
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, Inc. n=126
NCT04060199 COMPLETED
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
NS Pharma, Inc. n=77
NCT03532542 TERMINATED
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. n=171
NCT04632940 TERMINATED
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
Kyntra Bio n=73
NCT04371666 TERMINATED
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
Kyntra Bio n=98
NCT02851797 COMPLETED
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
Italfarmaco n=179
NCT03354039 COMPLETED
Tamoxifen in Duchenne Muscular Dystrophy
University Hospital, Basel, Switzerland n=93
NCT01603407 COMPLETED
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
University of Rochester n=196
NCT02814019 TERMINATED
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
Santhera Pharmaceuticals n=255
NCT03603288 TERMINATED
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
Santhera Pharmaceuticals n=161
NCT01183767 COMPLETED
Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
Charite University, Berlin, Germany n=33
NCT03907072 TERMINATED
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
Wave Life Sciences Ltd. n=6
NCT03039686 COMPLETED
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Hoffmann-La Roche n=166
NCT01557400 COMPLETED
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
PTC Therapeutics n=94
NCT03917719 TERMINATED
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Catabasis Pharmaceuticals n=130
NCT02354352 COMPLETED
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
Ohio State University n=52
NCT03642145 WITHDRAWN
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
PTC Therapeutics
NCT01126697 COMPLETED
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
Cooperative International Neuromuscular Research Group n=63
NCT01995032 COMPLETED
L-citrulline and Metformin in Duchenne's Muscular Dystrophy
University Hospital, Basel, Switzerland n=47
NCT00839033 TERMINATED
Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
Assistance Publique - Hôpitaux de Paris n=14
Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only