Duchenne muscular dystrophy: trials for referral
105 open trials in the registries we read. Each row links to its registry record.
Registry data only. Frontlight does not judge whether a patient can take part: the trial team decides.
Where the open trials are
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105 trials shown
Early Phase 11 site
RecruitingChina
NCT07188012
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Shanghai Siponuoyin Biotechnology Co Ltd
Registry updated 5 Nov 2026
Registry record: ClinicalTrials.govPhase 24 sites
RecruitingGeorgia, New Zealand
NCT07704099
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Keros Therapeutics, Inc.
Registry updated 4 Nov 2026
Registry record: ClinicalTrials.govPhase 20 sites
Not yet recruiting
NCT07911995
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 Dystrophy
Grünenthal GmbH
Registry updated 3 Nov 2026
Registry record: ClinicalTrials.govPhase 1/22 sites
RecruitingUnited States
NCT07429240
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Precision BioSciences, Inc.
Registry updated 21 Oct 2026
Registry record: ClinicalTrials.govPhase 2/314 sites
RecruitingBelgium, Spain, France, United Kingdom
NCT07856329
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
Genethon
Registry updated 13 Oct 2026
Registry record: ClinicalTrials.govPhase 26 sites
By invitation onlyAustralia, United States
NCT06867107
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-101
Satellos Bioscience, Inc.
Registry updated 13 Oct 2026
Registry record: ClinicalTrials.govPhase 318 sites
RecruitingAustralia, Belgium, Germany, Spain, France, United Kingdom
NCT07587242
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.
Registry updated 13 Oct 2026
Registry record: ClinicalTrials.govPhase 21 site
RecruitingUnited States
NCT07766980
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
University of Florida
Registry updated 8 Oct 2026
Registry record: ClinicalTrials.govPhase 41 site
RecruitingUnited States
NCT07542314
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
Sarepta Therapeutics, Inc.
Registry updated 6 Oct 2026
Registry record: ClinicalTrials.govPhase 320 sites
RecruitingAustralia, Belgium, Canada, Germany, Spain, France
NCT07160634
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Solid Biosciences Inc.
Registry updated 28 Sept 2026
Registry record: ClinicalTrials.govPhase 248 sites
By invitation onlyAustralia, Belgium, Germany, Denmark, Spain, France
NCT06066580
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
Edgewise Therapeutics, Inc.
Registry updated 17 Sept 2026
Registry record: ClinicalTrials.govPhase 112 sites
RecruitingUnited States
NCT04626674
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 Cohort
Sarepta Therapeutics, Inc.
Registry updated 11 Sept 2026
Registry record: ClinicalTrials.govNo phase1 site
Not yet recruitingPakistan
NCT07787689
Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy
Muhammad Aamir Latif
Registry updated 26 Aug 2026
Registry record: ClinicalTrials.govPhase 11 site
RecruitingUnited States
NCT07172971
Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy
Vanderbilt University Medical Center
Registry updated 24 Aug 2026
Registry record: ClinicalTrials.govPhase 44 sites
RecruitingUnited States
NCT05412394
Once Weekly Infant Corticosteroid Trial for DMD
Anne M. Connolly
Registry updated 14 Aug 2026
Registry record: ClinicalTrials.govPhase 1/221 sites
RecruitingBelgium, Spain, France, United Kingdom, Ireland, Italy
NCT07038824
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Entrada Therapeutics, Inc.
Registry updated 13 Aug 2026
Registry record: ClinicalTrials.govPhase 1/221 sites
RecruitingBelgium, Germany, Spain, France, United Kingdom, Ireland
NCT07037862
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
Entrada Therapeutics, Inc.
Registry updated 13 Aug 2026
Registry record: ClinicalTrials.govPhase 26 sites
By invitation onlySpain, United Kingdom, Italy, Netherlands
NCT07573631
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
BioMarin Pharmaceutical
Registry updated 24 Jul 2026
Registry record: ClinicalTrials.govPhase 329 sites
RecruitingBelgium, Canada, Czechia, Germany, Spain, France
NCT05933057
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Italfarmaco
Registry updated 24 Jul 2026
Registry record: ClinicalTrials.govPhase 1/215 sites
RecruitingCanada, United Kingdom, Italy, United States
NCT06138639
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Solid Biosciences Inc.
Registry updated 8 Jul 2026
Registry record: ClinicalTrials.govPhase 217 sites
Not yet recruitingBelgium, Spain, United Kingdom, Italy, Netherlands
NCT07682129
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)
Entrada Therapeutics, Inc.
Registry updated 2 Jul 2026
Registry record: ClinicalTrials.govPhase 1/26 sites
RecruitingBelarus, Russia
NCT07673809
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
AO GENERIUM
Registry updated 29 Jun 2026
Registry record: ClinicalTrials.govNo phase1 site
Not yet recruitingBelgium
NCT07664124
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
Centre Hospitalier Universitaire de Liege
Registry updated 23 Jun 2026
Registry record: ClinicalTrials.govPhase 1/21 site
Not yet recruitingUnited States
NCT07642635
Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy
Vanderbilt University Medical Center
Registry updated 11 Jun 2026
Registry record: ClinicalTrials.govPhase 31 site
RecruitingUnited States
NCT07608432
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Dyne Therapeutics
Registry updated 27 May 2026
Registry record: ClinicalTrials.govPhase 21 site
RecruitingUnited States
NCT06290713
Vasodilator and Exercise Study for DMD (VASO-REx)
University of Florida
Registry updated 15 May 2026
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlySweden
NCT07543302
Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients?
taVNS AB
Registry updated 24 Apr 2026
Registry record: ClinicalTrials.govNo phase0 sites
Not yet recruiting
NCT07475754
A Study to Evaluate the Safety and Tolerability of Rituxan in Duchenne Muscular Dystrophy
Peking Union Medical College Hospital
Registry updated 16 Mar 2026
Registry record: ClinicalTrials.govPhase 23 sites
By invitation onlyUnited Kingdom, Jordan
NCT07209332
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Wave Life Sciences USA, Inc.
Registry updated 6 Mar 2026
Registry record: ClinicalTrials.govPhase 11 site
RecruitingUnited States
NCT06692426
Trial of Cell Based Therapy for DMD
Masonic Cancer Center, University of Minnesota
Registry updated 4 Mar 2026
Registry record: ClinicalTrials.gov
The same sites as a list
- United States36 open trials
- Belgium19 open trials
- Italy19 open trials
- France17 open trials
- United Kingdom17 open trials
- Spain15 open trials
- Netherlands12 open trials
- Türkiye12 open trials
- Germany10 open trials
- China6 open trials
- Poland6 open trials
- Australia4 open trials
- Canada4 open trials
- Denmark3 open trials
- South Korea3 open trials
- New Zealand2 open trials
- Israel2 open trials
- Pakistan2 open trials
- Ireland2 open trials
- Czechia2 open trials
- Sweden2 open trials
- Serbia2 open trials
- Egypt2 open trials
- Georgia1 open trial
- Russia1 open trial
- Belarus1 open trial
- Jordan1 open trial
- Taiwan1 open trial
- Japan1 open trial
- Hong Kong1 open trial
- Hungary1 open trial
- Romania1 open trial
- Slovenia1 open trial
- Austria1 open trial
- Puerto Rico1 open trial
- Greece1 open trial
Who treats this
Institutions with trials or publications in this disease (OpenAlex, registries). Institutions only, no people.
Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda
Milan, Italy
2 trials · 5 publications · ROR
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
11 trials · 30 publications · ROR
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
5 trials · 8 publications · ROR
What changed in the last 7 days
Registry facts, one per line, newest first.
Phase changed: Phase 1/2 → Phase 2/3
NCT07856329 · Phase 2/3 · Belgium, Spain, France, United Kingdom
Source: ClinicalTrials.govStatus changed: Recruiting → Not yet recruiting
NCT07682129 · Phase 2 · Belgium, Spain, United Kingdom, Italy, Netherlands
Source: ClinicalTrials.govStatus changed: Recruiting → By invitation only
NCT07573631 · Phase 2 · Spain, United Kingdom, Italy, Netherlands
Source: ClinicalTrials.govStatus changed: Completed → Running, not recruiting
NCT06280209 · Phase 1/2 · Spain, United Kingdom, Italy, Netherlands
Source: ClinicalTrials.govStatus changed: Recruiting → By invitation only
NCT05967351 · Phase 3 · Belgium, Germany, Spain, United Kingdom, Hong Kong, Italy
Source: ClinicalTrials.govStatus changed: Completed → Running, not recruiting
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
NCT04768062 · Phase 3 · Australia, Canada, Chile, China, Czechia, Spain
Source: ClinicalTrials.govStatus changed: Completed → Running, not recruiting
NCT03992430 · Phase 3 · Switzerland, Colombia, Czechia, Germany, Denmark, Spain
Source: ClinicalTrials.govOpened for recruitment
NCT07682129 · Phase 2 · Belgium, Spain, United Kingdom, Italy, Netherlands
Source: ClinicalTrials.gov
Results card
Trials that finished or stopped in the last 3 years, with the reason the registry gives and linked publications.
No phaseEnded 30 Sept 2026
Phase 2Ended 7 Sept 2026
Completed244 participants
NCT05291091
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
Phase 3Ended 18 Jun 2026
Completed104 participants
NCT04587908
A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
Phase 3Ended 10 Feb 2026
Stopped early265 participants
NCT01247207
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
Reason given: Sponsor decision
Phase 2Ended 23 Jan 2026
No phaseEnded 11 Jan 2026
No phaseEnded 31 Dec 2025
No phaseEnded 12 Dec 2025
No phaseEnded 1 Dec 2025
Stopped early3 participants
NCT07254988
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Reason given: Terminated since further recruitment proved difficult
No phaseEnded 10 Nov 2025
Completed26 participants
NCT07437378
Relation Between Muscle Architecture and Functional Ability in Children With Duchenne Muscular Dystrophy
Phase 3Ended 16 Oct 2025
Completed228 participants
NCT02500381
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
No phaseEnded 12 Oct 2025
Completed50 participants
NCT07338812
Virtual Reality and Whole Body Vibration on Muscle Strength and Balance in Children With DMD
Phase 1Ended 10 Oct 2025
Stopped early5 participants
NCT06241950
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
Reason given: Study is being terminated due to a business decision.
Phase 3Ended 6 Oct 2025
Completed144 participants
NCT05066633
Metoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD)
Phase 2Ended 3 Oct 2025
Stopped early10 participants
NCT05429372
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
Reason given: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003
Phase 3Ended 24 Sept 2025
Stopped early7 participants
NCT05689164
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
Reason given: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
Phase 2/3Ended 8 Sept 2025
Phase 2Ended 4 Sept 2025
Phase 2Ended 28 Aug 2025
Stopped early7 participants
NCT06079736
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
Reason given: PGN-EDO51 development terminated by Sponsor
Phase 2Ended 18 Aug 2025
Show more
Phase 1Ended 5 Aug 2025
Stopped early3 participants
NCT06597656
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
Reason given: Study is being terminated due to a business decision.
No phaseEnded 31 Jul 2025
Completed28 participants
NCT06861270
Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France
Phase 1Ended 28 Jul 2025
Stopped early23 participants
NCT03362502
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
Reason given: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
Phase 1/2Ended 1 Jul 2025
Completed3 participants
NCT04240314
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
No phaseEnded 30 Jun 2025
Completed17 participants
NCT05833633
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
Phase 2Ended 28 May 2025
Withdrawn before start0 participants
NCT06833931
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Reason given: Global PGN-EDO51 development voluntarily discontinued by Sponsor
No phaseEnded 1 May 2025
Early Phase 1Ended 28 Apr 2025
Completed77 participants
NCT06565208
First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort
No phaseEnded 31 Mar 2025
Completed316 participants
NCT02369731
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
No phaseEnded 13 Mar 2025
No phaseEnded 21 Feb 2025
Phase 2Ended 14 Jan 2025
Completed1 participant
NCT05938023
A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in nonambulatory participants with Duchenne Muscular Dystrophy
No phaseEnded 31 Dec 2024
Completed38 participants
NCT04529707
Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy
Phase 1/2Ended 25 Nov 2024
Completed70 participants
NCT05670730
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
Phase 2Ended 6 Nov 2024
Phase 3Ended 25 Oct 2024
Completed126 participants
NCT05096221
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
- Three-Year Follow-Up of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Long-Term Phase 3 EMBARK Trial Results. · Neurology and therapy, 2026
- Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial. · Neurology and therapy, 2026
Phase 1Ended 16 Oct 2024
Completed18 participants
2024-513845-36-00
Evaluation of vamorolone CYP3A4 induction on midazolam (a sensitive CYP3A4 substrate) pharmacokinetics
No phaseEnded 30 Sept 2024
Completed50 participants
NCT05280730
Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
Phase 2Ended 16 Jul 2024
Completed54 participants
NCT05185622
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
- Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy. · Journal of neurology, 2026
- Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys. · Neurology, 2026
No phaseEnded 30 Jun 2024
No phaseEnded 30 Jun 2024
No phaseEnded 10 May 2024
Completed20 participants
NCT06295718
Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study
No phaseEnded Apr 2024
No phaseEnded 10 Mar 2024
Completed32 participants
NCT06379906
Upper Extremity Muscle Strength, Balance and Functional Skills in DMD
- The Impact of Trunk Control and Balance on Functional Skills in Ambulatory Children With Duchenne Muscular Dystrophy. · Journal of paediatrics and child health, 2026
Phase 1Ended 1 Mar 2024
Completed12 participants
NCT05160415
A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
No phaseEnded 29 Feb 2024
Completed16 participants
NCT06186310
Effectiveness of Aquatic Therapy in Children With Duchenne and Becker Muscular Dystrophy
- Effectiveness of aquatic therapy on balance and functionality in children with Duchenne and Becker muscular dystrophy : a prospective controlled pilot study. · Developmental neurorehabilitation, 2025
No phaseEnded 29 Feb 2024
No phaseEnded 1 Jan 2024
Phase 1/2Ended 31 Dec 2023
Completed2 participants
NCT03333590
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
Phase 3Ended 14 Dec 2023
Stopped early73 participants
NCT04632940
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
Reason given: Study did not meet its primary endpoint.
Not medical advice. fetched 10 Nov 2026