Disease landscape

Duchenne Muscular Dystrophy clinical trials

Get alerts when Duchenne Muscular Dystrophy trials change

131 active trials 60 recruiting 12 Phase 3 81 sponsors 19 changes in 30 days

Data updated daily from ClinicalTrials.gov, last refresh 2026-09-16

What changed in the last 30 days

2026-09-16
Primary completion pushed: 2026-09-11 → 2027-02-26
Enrollment closed, study ongoing
Trial sites expanded: 25 → 29 locations
Completion pushed: 2026-09-11 → 2027-02-26
NCT05996003 NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) · NS Pharma, Inc. · Phase 2
2026-08-28
Primary completion pushed: 2027-03-31 → 2027-08-31
Completion pushed: 2027-03-31 → 2027-08-31
NCT07542314 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne… · Sarepta Therapeutics, Inc. · Phase 4
2026-08-18
Trial completed
Primary completion moved earlier: 2027-05 → 2026-06-18
Completion moved earlier: 2027-05 → 2026-06-18
NCT04587908 A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD） · Taiho Pharmaceutical Co., Ltd. · Phase 3
2026-09-05
Primary completion pushed: 2026-08-26 → 2026-11-18
NCT06450639 A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy… · Hoffmann-La Roche · Phase 2
2026-09-04
Trial completed
Final enrollment: 8 participants (planned 30)
NCT06363357 The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements… · Seoul National University Hospital
2026-09-12
Trial sites expanded: 7 → 12 locations
NCT04626674 A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From… · Sarepta Therapeutics, Inc. · Phase 1
2026-08-29
Completion pushed: 2026-12 → 2027-03
NCT05126758 A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne… · Capricor Inc. · Phase 3
2026-08-20
Trial sites expanded: 1 → 4 locations
NCT07092540 The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First… · University of Rochester
2026-09-06
Trial status changed: Recruiting → Unknown
NCT06539169 FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · xCures
2026-09-06
Trial status changed: Recruiting → Unknown
NCT06581887 Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies · University College, London
2026-09-06
Trial status changed: Recruiting → Unknown
NCT06574919 Clinical and Functional Assessment of Patients With Inherited Non-Duchenne Myopathies in… · Sohag University
2026-09-06
Trial status changed: Not Yet Recruiting → Unknown
NCT06540365 Applying the Pathways and Resources for Engagement and Participation Protocol Among… · University of Haifa

New sponsor entrants, last 90 days sponsors with their first trial in this area

First postedTrialSponsorPhase
2026-08-26 NCT07787689 Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne… Muhammad Aamir Latif new
2026-07-15 NCT07704099 Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy Keros Therapeutics, Inc. new Phase 2
2026-06-29 NCT07673809 A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in… AO GENERIUM new Phase 1/2
2026-06-23 NCT07664124 Digital Monitoring of Upper Limb Function in Non-Ambulant DMD Centre Hospitalier Universitaire de Liege new

Active trials by phase

Early Phase 1
6
Phase 1
6
Phase 1/2
12
Phase 2
19
Phase 2/3
3
Phase 3
9
Phase 4
4
Not charted: 72 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

Active trials top 25 of 131

NCT IDTitleSponsorStatusUpdated
Phase 4
NCT05412394 Once Weekly Infant Corticosteroid Trial for DMD Anne M. Connolly Recruiting 2026-08-14
NCT07542314 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne… Sarepta Therapeutics, Inc. Not Yet Recruiting 2026-08-27
NCT06713135 A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With… Santhera Pharmaceuticals Active, Not Recruiting 2026-04-23
NCT04687020 Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical… NS Pharma, Inc. Active, Not Recruiting 2022-10-19
Phase 3
NCT07587242 A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as… Avidity Biosciences, Inc. Recruiting 2026-09-15
NCT07160634 A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy… Solid Biosciences Inc. Recruiting 2026-08-13
NCT05933057 Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne… Italfarmaco Recruiting 2026-07-24
NCT07608432 Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered… Dyne Therapeutics Recruiting 2026-05-27
NCT05126758 A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne… Capricor Inc. Active, Not Recruiting 2026-08-28
NCT04281485 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne… Pfizer Active, Not Recruiting 2026-07-22
NCT05881408 A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene… Sarepta Therapeutics, Inc. Active, Not Recruiting 2026-05-22
NCT03992430 A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With… Sarepta Therapeutics, Inc. Active, Not Recruiting 2026-02-27
NCT05967351 A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec… Sarepta Therapeutics, Inc. Enrolling by Invitation 2026-02-06
Phase 2/3
NCT03373968 Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study Italfarmaco Recruiting 2026-01-21
NCT05693142 AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy… REGENXBIO Inc. Active, Not Recruiting 2026-07-21
NCT05195775 Tadalafil as Adjuvant Therapy for DMD University of Florida Active, Not Recruiting 2025-02-27
Phase 2
NCT07287189 Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients Satellos Bioscience, Inc. Recruiting 2026-08-14
NCT06290713 Vasodilator and Exercise Study for DMD (VASO-REx) University of Florida Recruiting 2026-05-15
NCT06769633 Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to… Italfarmaco Recruiting 2025-07-10
NCT05996003 NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) NS Pharma, Inc. Active, Not Recruiting 2026-09-15
NCT06128564 A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene… Hoffmann-La Roche Active, Not Recruiting 2026-09-09
NCT06450639 A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy… Hoffmann-La Roche Active, Not Recruiting 2026-09-04
NCT06579352 Study of UC-MSC in DMD Patients MED Institute Inc. Active, Not Recruiting 2026-08-14
NCT07704099 Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy Keros Therapeutics, Inc. Not Yet Recruiting 2026-08-10
NCT06244082 Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With… Avidity Biosciences, Inc. Active, Not Recruiting 2026-07-30
See all 131 active Duchenne Muscular Dystrophy trials with filters →

Top sponsors by active trials in this area

Sarepta Therapeutics, Inc. (7) · Lokman Hekim University (6) · Vanderbilt University Medical Center (5) · University of Florida (4) · Centre Hospitalier Universitaire de Liege (3) · Entrada Therapeutics, Inc. (3) · Italfarmaco (3) · NS Pharma, Inc. (3) · Peking Union Medical College Hospital (3) · REGENXBIO Inc. (3)

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