| Not Yet Recruiting | A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children NCT07511920 | West China Second University Hospital | — |
| Recruiting | Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases NCT06833489 | Assistance Publique Hopitaux De Marseille | N/A |
| Recruiting | Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders NCT06147414 | Assistance Publique - Hôpitaux de Paris | — |
| Recruiting | The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in P NCT06363357 | Seoul National University Hospital | N/A |
| Terminated | Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy NCT05429372 | Pfizer | Phase 2 |
| Active Not Recruiting | A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy NCT05126758 | Capricor Inc. | Phase 3 |
| Unknown | Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations NCT05833633 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Unknown | Characterization of DupEx2 Duchenne Muscular Dystrophy NCT06337669 | IRCCS San Raffaele | — |
| Recruiting | The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in NCT05066633 | Medical University of Gdansk | Phase 3 |
| Completed | Regular Physical Exercise in Duchenne Muscular Dystrophy NCT03963453 | Haukeland University Hospital | — |
| Recruiting | A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP- NCT04626674 | Sarepta Therapeutics, Inc. | Phase 1 |
| Active Not Recruiting | A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dyst NCT03992430 | Sarepta Therapeutics, Inc. | Phase 3 |
| Completed | Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular NCT05209087 | Hacettepe University | — |
| Completed | Safety Study of BLS-M22 in Healthy Volunteers NCT03789734 | BioLeaders Corporation | Phase 1 |
| Terminated | An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP NCT03675126 | Sarepta Therapeutics, Inc. | Phase 1 / Phase 2 |
| Terminated | Spironolactone Versus Prednisolone in DMD NCT03777319 | Kevin Flanigan | Phase 1 |
| Completed | A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne M NCT03769116 | Sarepta Therapeutics, Inc. | Phase 1 / Phase 2 |
| Completed | Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy NCT03703882 | Catabasis Pharmaceuticals | Phase 3 |
| Terminated | A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy NCT03400852 | Mallinckrodt ARD LLC | Phase 2 |
| Completed | Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomog NCT03490214 | University of Erlangen-Nürnberg Medical School | N/A |
| Completed | The Effects of Trunk Exercises on Upper Extremity and Respiratory Functions in DMD NCT04669847 | Gokce Yagmur Gunes Gencer | N/A |
| Completed | A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy NCT03406780 | Capricor Inc. | Phase 2 |
| Completed | A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirse NCT03375255 | Sarepta Therapeutics, Inc. | Phase 1 |
| Completed | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy NCT03179631 | PTC Therapeutics | Phase 3 |
| Completed | User-centred Assistive System for Arm Functions in Neuromuscular Subjects NCT03127241 | Politecnico di Milano | N/A |
| Completed | Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD NCT02834650 | Stanford University | N/A |
| Completed | Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy NCT02439216 | Catabasis Pharmaceuticals | Phase 1 / Phase 2 |
| Completed | Rimeporide in Patients With Duchenne Muscular Dystrophy NCT02710591 | EspeRare Foundation | Phase 1 |
| Completed | Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) NCT02369731 | PTC Therapeutics | — |
| Completed | Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet NCT02383511 | Summit Therapeutics | Phase 1 |
| Completed | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy NCT02286947 | Sarepta Therapeutics, Inc. | Phase 2 |
| Terminated | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy NCT02090959 | PTC Therapeutics | Phase 3 |
| Completed | Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy NCT02195999 | University of Florida | — |
| Terminated | A Study of Tadalafil for Duchenne Muscular Dystrophy NCT01865084 | Eli Lilly and Company | Phase 3 |
| Unknown | Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscula NCT01918384 | Kobe University | Phase 2 |
| Completed | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) NCT01826487 | PTC Therapeutics | Phase 3 |
| Completed | Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients NCT01826422 | Coordinación de Investigación en Salud, Mexico | N/A |
| Completed | Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dyst NCT01506518 | University of North Carolina, Chapel Hill | — |
| Completed | Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) NCT01027884 | Santhera Pharmaceuticals | Phase 3 |
| Completed | Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy NCT00296621 | Assistance Publique - Hôpitaux de Paris | Phase 2 |
| Completed | Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy NCT00243789 | Cooperative International Neuromuscular Research Group | Phase 1 / Phase 2 |
| Completed | Pentoxifylline in Duchenne Muscular Dystrophy NCT00102453 | Cooperative International Neuromuscular Research Group | Phase 1 / Phase 2 |
| Completed | KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy NCT00033813 | Cooperative International Neuromuscular Research Group | Phase 2 |
| Completed | An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy NCT00033189 | Cooperative International Neuromuscular Research Group | Phase 2 |
| Completed | Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy NCT00016653 | Cooperative International Neuromuscular Research Group | Phase 2 / Phase 3 |
| Available | Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping NCT07250737 | Avidity Biosciences, Inc. | — |
| Completed | A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and NCT00018109 | National Center for Research Resources (NCRR) | Phase 3 |
| Approved For Marketing | The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation NCT04337112 | NS Pharma, Inc. | — |