| Enrolling By Invitation | Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy NCT07209332 | Wave Life Sciences Ltd. | Phase 2 |
| Recruiting | Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients NCT07287189 | Satellos Bioscience, Inc. | Phase 2 |
| Recruiting | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) NCT07160634 | Solid Biosciences Inc. | Phase 3 |
| Recruiting | A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Amb NCT06817382 | Insmed Gene Therapy LLC | Phase 1 |
| Recruiting | Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy NCT06402942 | Başak Çağla Arslan | N/A |
| Active Not Recruiting | A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) NCT06450639 | Hoffmann-La Roche | Phase 2 |
| Recruiting | Trial of Cell Based Therapy for DMD NCT06692426 | Masonic Cancer Center, University of Minnesota | Phase 1 |
| Enrolling By Invitation | Investigation of the Relationship Between Executive Functions and Occupational Performance of Children With Du NCT06868784 | Lokman Hekim University | — |
| Enrolling By Invitation | Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies NCT06363526 | University of Malaga | N/A |
| Recruiting | Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old NCT06769633 | Italfarmaco | Phase 2 |
| Not Yet Recruiting | Evaluation of Brain Dysfunction in Patients with Duchene Muscular Dystrophy NCT06732011 | Assiut University | — |
| Not Yet Recruiting | The U.K. NorthStar Clinical Network NCT06711692 | University College, London | — |
| Active Not Recruiting | A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dyst NCT06713135 | Santhera Pharmaceuticals | Phase 4 |
| Withdrawn | Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP) NCT05558813 | Assistance Publique - Hôpitaux de Paris | N/A |
| Not Yet Recruiting | Development of a Registry to Assess Natural History in Duchenne Muscular Dystrophy NCT06579859 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Recruiting | Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrop NCT06564974 | Catalyst Pharmaceuticals, Inc. | — |
| Terminated | A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Followi NCT06597656 | Sarepta Therapeutics, Inc. | Phase 1 |
| Recruiting | NS-050/NCNP-03 in Boys With DMD (Meteor50) NCT06053814 | NS Pharma, Inc. | Phase 1 / Phase 2 |
| Completed | First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort NCT06565208 | Satellos Bioscience, Inc. | EARLY_Phase 1 |
| Not Yet Recruiting | Applying the Pathways and Resources for Engagement and Participation Protocol Among People With Muscles Dystro NCT06540365 | University of Haifa | N/A |
| Recruiting | Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim NCT06093100 | Vanderbilt University Medical Center | — |
| Recruiting | FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases NCT06539169 | xCures | — |
| Recruiting | Vasodilator and Exercise Study for DMD (VASO-REx) NCT06290713 | University of Florida | Phase 2 |
| Recruiting | Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France NCT06861270 | Assistance Publique - Hôpitaux de Paris | — |
| Enrolling By Invitation | Long Term Follow-up for RGX-202 NCT06491927 | REGENXBIO Inc. | — |
| Recruiting | A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) NCT06138639 | Solid Biosciences Inc. | Phase 1 / Phase 2 |
| Completed | Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Res NCT07172737 | Izmir Katip Celebi University | N/A |
| Recruiting | Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases NCT06756633 | Lokman Hekim University | — |
| Active Not Recruiting | Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With NCT06100887 | Edgewise Therapeutics, Inc. | Phase 2 |
| Recruiting | Wearable Technology to Evaluate Hyperglycemia and HRV in DMD NCT06124196 | Vanderbilt University Medical Center | — |
| Unknown | Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy NCT06328725 | ENCell | Phase 1 / Phase 2 |
| Recruiting | NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) NCT05996003 | NS Pharma, Inc. | Phase 2 |
| Recruiting | Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy NCT05933057 | Italfarmaco | Phase 3 |
| Enrolling By Invitation | An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duche NCT06270719 | Sarepta Therapeutics, Inc. | — |
| Active Not Recruiting | A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of J NCT06114056 | West China Hospital | Phase 1 |
| Terminated | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) F NCT06241950 | Sarepta Therapeutics, Inc. | Phase 1 |
| Active Not Recruiting | Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 NCT06244082 | Avidity Biosciences, Inc. | Phase 2 |
| Terminated | A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment NCT06079736 | PepGen Inc | Phase 2 |
| Recruiting | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Par NCT06280209 | BioMarin Pharmaceutical | Phase 1 / Phase 2 |
| Unknown | Telerehabilitation-based Motor Imagery Training in Children With Duchenne Muscular Dystrophy NCT06109103 | Hacettepe University | N/A |
| Active Not Recruiting | A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Un NCT06128564 | Hoffmann-La Roche | Phase 2 |
| Completed | Upper Extremity Muscle Strength, Balance and Functional Skills in DMD NCT06379906 | Halic University | — |
| Completed | NSAA NON-Interventional Study Protocol NCT06054971 | Red Nucleus Enterprise Solutions, LLC | — |
| Enrolling By Invitation | A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous NCT05967351 | Sarepta Therapeutics, Inc. | Phase 3 |
| Unknown | Effect of Kinesiology Taping on Head and Trunk Control in Children With Duchenne Muscular Dystrophy NCT05967793 | Egyptian Chinese University | N/A |
| Unknown | Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Va NCT06295718 | Sahra Şirvan | — |
| Completed | Validity and Reliability of the 6 Minute Pegboard Ring Test NCT06174025 | Çağtay Maden | — |
| Enrolling By Invitation | Psychoeducation Program for Parents of Children with Duchenne Muscular Dystrophy NCT06412328 | Lokman Hekim University | N/A |
| Active Not Recruiting | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) i NCT05881408 | Sarepta Therapeutics, Inc. | Phase 3 |
| Enrolling By Invitation | Follow-up Study on Female Carriers With DMD Gene Variants NCT05715957 | Rigshospitalet, Denmark | — |
| Terminated | A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy NCT05938023 | Percheron Therapeutics | Phase 2 |
| Unknown | Bicycle Training on Ventilatory Functions in Duchenne Muscular Dystrophy NCT05849688 | Beni-Suef University | N/A |
| Unknown | Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy NCT05249361 | Samsung Medical Center | — |
| Unknown | Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy NCT05753462 | Sqy Therapeutics | Phase 1 / Phase 2 |
| Unknown | DMD- Interactive Virtual Reality Study NCT06274983 | Sheffield Children's NHS Foundation Trust | N/A |
| Terminated | A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular D NCT05689164 | Pfizer | Phase 3 |
| Recruiting | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) NCT05693142 | REGENXBIO Inc. | Phase 2 / Phase 3 |
| Recruiting | AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD NCT05683379 | REGENXBIO Inc. | — |
| Unknown | Motor Imagery on Children With DMD on Gait and Balance Functions NCT05601986 | Gülsena Utku | N/A |
| Unknown | Physical Activity Level and Cognitive Functions in Children With Duchenne Muscular Dystrophy NCT05641805 | Muş Alparslan University | — |
| Completed | Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutation NCT05670730 | Avidity Biosciences, Inc. | Phase 1 / Phase 2 |
| Active Not Recruiting | Hydrotherapy in Duchenne Muscular Dystrophy (DMD) NCT06445985 | Lancashire Teaching Hospitals NHS Foundation Trust | N/A |
| Completed | Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment NCT05549999 | Hacettepe University | — |
| Active Not Recruiting | A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) NCT05540860 | Edgewise Therapeutics, Inc. | Phase 2 |
| Completed | Evaluation of Home Based Assessments on Participants With DMD NCT05657938 | Solid Biosciences Inc. | — |
| Completed | Dual Task in Duchenne Muscular Dystrophy NCT05575648 | Hacettepe University | N/A |
| Completed | Duchenne Muscular Dystrophy Video Assessment Registry NCT05712447 | The Emmes Company, LLC | — |
| Unknown | Treatment of a Single Patient With CRD-TMH-001 NCT05514249 | Cure Rare Disease, Inc | Phase 1 |
| Recruiting | phenotypeS in Non Ambulant Duchenne Muscular Dystrophy NCT06366815 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Completed | Postural and Anthropometric Properties of Foot and Ankle of Patients With DMD NCT05436210 | Hacettepe University | — |
| Completed | Motor Imagery in Duchenne Muscular Dystrophy NCT05559710 | Hacettepe University | — |
| Unknown | Schulze Muscular Dystrophy Ability Clinical Study NCT05409079 | AbiliTech Medical Inc. | N/A |
| Unknown | Neuropsychological Profiles of Children With Duchenne Muscular Dystrophy and Its Effects on Motor Functions NCT05661071 | Hacettepe University | — |
| Completed | Bicycle Ergometer Training in Duchenne Muscular Dystrophy NCT05330195 | Hacettepe University | N/A |
| Completed | A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (D NCT05185622 | Santhera Pharmaceuticals | Phase 2 |
| Completed | Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy NCT05280730 | Virginia Commonwealth University | — |
| Completed | Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) NCT05338099 | ENCell | Phase 1 |
| Active Not Recruiting | Tadalafil as Adjuvant Therapy for DMD NCT05195775 | University of Florida | Phase 2 / Phase 3 |
| Unknown | Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices NCT05110885 | Bahçeşehir University | — |
| Completed | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) i NCT05096221 | Sarepta Therapeutics, Inc. | Phase 3 |
| Completed | Brain Involvement in Dystrophinopathies Part 2 NCT04668716 | Great Ormond Street Hospital for Children NHS Foundation Trust | — |
| Unknown | Comprehensive Study of Duchenne Muscular Dystrophy at Sohag University Hospital NCT05029232 | Sohag University | N/A |
| Completed | Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy NCT05464446 | Lokman Hekim University | — |
| Completed | Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Rec NCT04335942 | Assistance Publique - Hôpitaux de Paris | N/A |
| Recruiting | Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) NCT04906460 | Wave Life Sciences Ltd. | Phase 1 / Phase 2 |
| Recruiting | CureDuchenne Link®: A Resource for Research NCT04972604 | CureDuchenne | — |
| Completed | Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With D NCT04956289 | NS Pharma, Inc. | Phase 2 |
| Active Not Recruiting | Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) NCT04687020 | NS Pharma, Inc. | Phase 4 |
| Recruiting | Once Weekly Infant Corticosteroid Trial for DMD NCT05412394 | Anne M. Connolly | Phase 4 |
| Unknown | Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) NCT04768062 | NS Pharma, Inc. | Phase 3 |
| Unknown | The Effect of Telerehabilitation of Patients With Duchenne Muscular Dystrophy NCT04782440 | Marmara University | N/A |
| Completed | Brain Involvement in Dystrophinopathies Part 1 NCT04583917 | Great Ormond Street Hospital for Children NHS Foundation Trust | — |
| Terminated | Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambu NCT04632940 | Kyntra Bio | Phase 3 |
| Completed | Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy NCT04529707 | University of Pittsburgh | N/A |
| Completed | Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD) NCT05564962 | Hacettepe University | — |
| Completed | Telerehabilitation in Duchenne Muscular Dystrophy NCT05305976 | Karadeniz Technical University | N/A |
| Completed | Remote Physiotherapy to Protect Physical Health in Duchenne Muscular Dystrophy NCT06103006 | Lokman Hekim University | N/A |
| Active Not Recruiting | Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy NCT04281485 | Pfizer | Phase 3 |
| Active Not Recruiting | A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD） NCT04587908 | Taiho Pharmaceutical Co., Ltd. | Phase 3 |
| Terminated | An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy NCT04708314 | Rare Disease Research, LLC | Phase 4 |
| Recruiting | Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy NCT01484678 | University of Florida | — |
| Completed | Physical Activity Level of Norwegian Boys with Duchenne Muscular Dystrophy NCT03947112 | Haukeland University Hospital | — |
| Terminated | Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duch NCT04371666 | Kyntra Bio | Phase 3 |
| Active Not Recruiting | Open-label Extension of the HOPE-2 Trial NCT04428476 | Capricor Inc. | Phase 2 |
| Recruiting | Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study NCT05982119 | Centre Hospitalier Universitaire de Liege | N/A |
| Active Not Recruiting | Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy NCT04433234 | Daiichi Sankyo Co., Ltd. | Phase 2 |
| Completed | Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) NCT04060199 | NS Pharma, Inc. | Phase 3 |
| Completed | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subj NCT04179409 | Kevin Flanigan | Phase 2 |
| Completed | Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrop NCT05244395 | Hacettepe University | N/A |
| Completed | AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. NCT04240314 | Megan Waldrop | Phase 1 / Phase 2 |
| Recruiting | Natural History of Duchenne Muscular Dystrophy NCT03882827 | Genethon | — |
| Completed | Exploratory Study of NS-089/NCNP-02 in DMD NCT04129294 | National Center of Neurology and Psychiatry, Japan | Phase 1 / Phase 2 |
| Recruiting | Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy NCT05016908 | Massachusetts General Hospital | — |
| Completed | Wearable Technology to Assess Gait Function in SMA and DMD NCT04193085 | Columbia University | — |
| Terminated | Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duc NCT03907072 | Wave Life Sciences Ltd. | Phase 2 / Phase 3 |
| Completed | A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy NCT03760029 | Pfizer | N/A |
| Recruiting | A Registered Cohort Study on Duchenne Muscular Dystrophy NCT04012671 | Ning Wang, MD., PhD. | — |
| Terminated | A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dy NCT03985878 | Sarepta Therapeutics, Inc. | Phase 2 |
| Terminated | Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in P NCT04004065 | Sarepta Therapeutics, Inc. | Phase 2 |
| Completed | The Burden of Access in Duchenne Muscular Dystrophy in the US NCT03951675 | University of Florida | — |
| Unknown | Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy NCT03936894 | Children's National Research Institute | Phase 1 / Phase 2 |
| Completed | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) NCT03796637 | PTC Therapeutics | Phase 2 |
| Completed | Aerobic Exercise in Duchenne Muscular Dystrophy NCT04173234 | Hacettepe University | N/A |
| Unknown | The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystroph NCT04287582 | Hacettepe University | — |
| Completed | Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease NCT04120168 | Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition | — |
| Completed | Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dys NCT05274555 | Dokuz Eylul University | — |
| Terminated | An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy NCT03917719 | Catabasis Pharmaceuticals | Phase 3 |
| Unknown | Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Bi NCT03689660 | Merve Kurt | N/A |
| Unknown | Bisoprolol in DMD Early Cardiomyopathy NCT03779646 | Peking Union Medical College Hospital | Phase 2 / Phase 3 |
| Enrolling By Invitation | A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice NCT06606340 | Sarepta Therapeutics, Inc. | — |
| Completed | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) NCT03648827 | PTC Therapeutics | Phase 2 |
| Enrolling By Invitation | Parent and Infant Inter(X)Action Intervention (PIXI) NCT03836300 | RTI International | N/A |
| Withdrawn | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) NCT03642145 | PTC Therapeutics | Phase 3 |
| Enrolling By Invitation | Early Check: Expanded Screening in Newborns NCT03655223 | RTI International | — |
| Completed | Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families NCT03680365 | Jett Foundation, Inc. | — |
| Unknown | The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :Peds NCT03513367 | University Hospital, Toulouse | — |
| Terminated | Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD NCT03531788 | Roxanna Marie Bendixen | N/A |
| Completed | Biomarker for Duchenne Muscular Dystrophy NCT02994030 | CENTOGENE GmbH Rostock | — |
| Terminated | Safety and Efficacy of P-188 NF in DMD Patients NCT03558958 | Phrixus Pharmaceuticals, Inc. | Phase 2 |
| Unknown | Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device NCT03611244 | Seoul National University Hospital | N/A |
| Terminated | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy NCT03532542 | Sarepta Therapeutics, Inc. | Phase 3 |
| Terminated | Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) NCT03603288 | Santhera Pharmaceuticals | Phase 3 |
| Completed | A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) NCT03439670 | ReveraGen BioPharma, Inc. | Phase 2 |
| Completed | Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) NCT06304064 | Capricor Inc. | Phase 2 |
| Completed | Tamoxifen in Duchenne Muscular Dystrophy NCT03354039 | University Hospital, Basel, Switzerland | Phase 3 |
| 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 | UTSW HP [13-C] Pyruvate Injection in HCM NCT03057002 | University of Texas Southwestern Medical Center | — |
| Withdrawn | Relationship Between PFTs and Pdi in DMD NCT03335384 | University of Minnesota | N/A |
| Unknown | Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dyst NCT03507530 | Canan İpek | — |
| Completed | Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy NCT03589612 | Hacettepe University | — |
| Completed | Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy NCT03508947 | Wave Life Sciences Ltd. | Phase 1 |
| Terminated | A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy NCT03362502 | Pfizer | Phase 1 |
| Completed | A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participant NCT03375164 | Sarepta Therapeutics, Inc. | Phase 1 / Phase 2 |
| Unknown | Prognostic Factors Affecting Duchenne Muscular Dystrophy NCT03372655 | Assiut University | — |
| Active Not Recruiting | Microdystrophin Gene Transfer Study in Adolescents and Children With DMD NCT03368742 | Solid Biosciences Inc. | Phase 1 / Phase 2 |
| Completed | Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy NCT03333590 | Kevin Flanigan | Phase 1 / Phase 2 |
| Recruiting | Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study NCT03373968 | Italfarmaco | Phase 2 / Phase 3 |
| Completed | Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD) NCT03319030 | Ann & Robert H Lurie Children's Hospital of Chicago | — |
| Completed | Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping NCT03218995 | Sarepta Therapeutics, Inc. | Phase 2 |
| Completed | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenn NCT03039686 | Hoffmann-La Roche | Phase 2 / Phase 3 |
| Completed | Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) NCT03167255 | NS Pharma, Inc. | Phase 2 |
| Unknown | Duchenne Muscular Dystrophy Heart Study NCT03443115 | Hôpital Cochin | — |
| Completed | Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Co NCT03529240 | Hacettepe University | N/A |
| Completed | The Effect of Kinesiology Taping on Balance in Duchenne Muscular Dystrophy NCT03541070 | Hacettepe University | N/A |
| Completed | Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular D NCT02851797 | Italfarmaco | Phase 3 |
| Completed | Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy NCT03521271 | Hacettepe University | — |
| Completed | Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy NCT03589573 | Hacettepe University | — |
| Completed | Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) NCT03038399 | ReveraGen BioPharma, Inc. | Phase 2 |
| Completed | Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance NCT03534349 | Hacettepe University | — |
| Completed | Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy NCT03552874 | Hacettepe University | — |
| Completed | Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) NCT02740972 | NS Pharma, Inc. | Phase 2 |
| Completed | Treatment Effect of Tamoxifen on Patients With DMD NCT02835079 | Hadassah Medical Organization | Phase 1 |
| Completed | Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents NCT02964377 | Craig McDonald, MD | Phase 1 / Phase 2 |
| Terminated | An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy NCT02907619 | Pfizer | Phase 2 |
| Completed | Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD NCT02500381 | Sarepta Therapeutics, Inc. | Phase 3 |
| Completed | An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) NCT02760277 | ReveraGen BioPharma, Inc. | Phase 2 |
| Completed | Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy NCT02819557 | PTC Therapeutics | Phase 2 |
| Completed | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) NCT02760264 | ReveraGen BioPharma, Inc. | Phase 2 |
| Terminated | Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular D NCT02858362 | Summit Therapeutics | Phase 2 |
| Active Not Recruiting | Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD NCT02972580 | Nationwide Children's Hospital | — |
| Completed | A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy NCT02752048 | Taiho Pharmaceutical Co., Ltd. | Phase 2 |
| Terminated | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) NCT02958202 | BioMarin Pharmaceutical | Phase 2 |
| Withdrawn | Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 NCT02704325 | Kevin Flanigan | Phase 1 / Phase 2 |
| Completed | Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases NCT03786913 | Benha University | — |
| Completed | Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy NCT02891434 | University of Sao Paulo | N/A |
| Completed | HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) NCT02485938 | Capricor Inc. | Phase 2 |
| Terminated | Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) NCT02606136 | Kyntra Bio | Phase 2 |
| Completed | Virtual Reality in Individuals With Duchenne Muscular Dystrophy NCT03002298 | University of Sao Paulo | N/A |
| Completed | Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy NCT02571205 | Newcastle-upon-Tyne Hospitals NHS Trust | — |
| Completed | Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) NCT02530905 | Sarepta Therapeutics, Inc. | Phase 1 |
| Completed | Study of DS-5141b in Patients With Duchenne Muscular Dystrophy NCT02667483 | Daiichi Sankyo Co., Ltd. | Phase 1 / Phase 2 |
| Unknown | Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy NCT03067831 | Stem Cells Arabia | Phase 1 / Phase 2 |
| Unknown | Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Pha NCT02484560 | University of Gaziantep | Phase 1 |
| Completed | Strength Training in Duchenne Muscular Dystrophy NCT02421523 | University of Florida | N/A |
| Terminated | HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02 NCT02525302 | Akashi Therapeutics | Phase 2 |
| Completed | Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy NCT02354352 | Ohio State University | Phase 3 |
| Completed | Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular NCT02376816 | Jerry R. Mendell | Phase 1 |
| Recruiting | Biomarker Development for Muscular Dystrophies NCT05019625 | Massachusetts General Hospital | — |
| Completed | Phase I/II Study of SRP-4053 in DMD Patients NCT02310906 | Sarepta Therapeutics, Inc. | Phase 1 / Phase 2 |
| Completed | Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dy NCT02354781 | Jerry R. Mendell | Phase 1 / Phase 2 |
| Completed | A Device for Rapid, Painless, Bedside Muscle Evaluation of Children NCT02340923 | Skulpt, Inc. | — |
| Completed | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrop NCT02251600 | PTC Therapeutics | Phase 1 |
| Terminated | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) NCT02329769 | BioMarin Pharmaceutical | Phase 2 |
| Completed | An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort NCT02295748 | PTC Therapeutics | Phase 1 |
| Terminated | A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duch NCT02310763 | Pfizer | Phase 2 |
| Unknown | Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy NCT01834040 | Chaitanya Hospital, Pune | Phase 1 / Phase 2 |
| Completed | A Study of TAS-205 for Duchenne Muscular Dystrophy NCT02246478 | Taiho Pharmaceutical Co., Ltd. | Phase 1 |
| Unknown | Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy. NCT01834066 | Chaitanya Hospital, Pune | Phase 1 / Phase 2 |
| Terminated | European Home Mechanical Ventilation Registry NCT02315339 | ResMed | — |
| Completed | Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle NCT02168114 | University of Florida | N/A |
| Unknown | Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies NCT02109692 | University Hospital, Montpellier | N/A |
| Unknown | Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients NCT02196467 | CHU de Quebec-Universite Laval | Phase 1 / Phase 2 |
| Completed | Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy NCT02167217 | Washington University School of Medicine | Phase 2 |
| Completed | Heart Rate Variability in Duchenne Muscular Dystrophy During Computer Task NCT04607824 | University of Sao Paulo | — |
| Completed | 2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children NCT02418338 | University Hospital, Montpellier | N/A |
| Completed | Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy NCT02034305 | University of Pittsburgh | — |
| Completed | A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD) NCT02056808 | Summit Therapeutics | Phase 1 |
| Unknown | Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy NCT02285673 | Acibadem University | Phase 1 / Phase 2 |
| Terminated | Open Label Extension Study of HT-100 in Patients With DMD NCT01978366 | Processa Pharmaceuticals | Phase 2 |
| Completed | Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of L NCT01963897 | Oslo University Hospital | — |
| Completed | At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurolo NCT01931644 | Sanguine Biosciences | — |
| Terminated | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) NCT01957059 | BioMarin Pharmaceutical | Phase 1 / Phase 2 |
| Completed | Exploratory Study of NS-065/NCNP-01 in DMD NCT02081625 | National Center of Neurology and Psychiatry, Japan | Phase 1 |
| Completed | Duchenne Muscular Dystrophy Clinical Trial NCT01874275 | Alan Neuromedical Technologies, LLC | N/A |
| Terminated | Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrop NCT01847573 | Processa Pharmaceuticals | Phase 1 / Phase 2 |
| Completed | Stacking Exercises Aid the Decline in FVC and Sick Time NCT01999075 | Children's Hospital of Eastern Ontario | Phase 4 |
| Completed | Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy NCT01954940 | Children's Hospital of Eastern Ontario | N/A |
| Completed | Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy NCT04740554 | University of Sao Paulo | — |
| Terminated | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy NCT01826474 | BioMarin Pharmaceutical | Phase 1 / Phase 2 |
| Unknown | Upper Limb Assessment in Duchenne Muscular Dystrophy NCT02436720 | Catholic University of the Sacred Heart | — |
| Completed | Finding the Optimum Regimen for Duchenne Muscular Dystrophy NCT01603407 | University of Rochester | Phase 3 |
| Terminated | A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy. NCT01753804 | BioMarin Pharmaceutical | — |
| Unknown | Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping NCT01772043 | Cooperative International Neuromuscular Research Group | — |
| Active Not Recruiting | Pediatric Radio Frequency Coils Generic NCT01633866 | Children's Hospital Medical Center, Cincinnati | — |
| Completed | Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystroph NCT01557400 | PTC Therapeutics | Phase 3 |
| Completed | Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study NCT02780492 | University College, London | — |
| Completed | Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy NCT01491555 | Boston Children's Hospital | — |
| Completed | PDE Inhibitors in DMD Study (Acute Dosing Study) NCT01580501 | Cedars-Sinai Medical Center | Phase 1 |
| Completed | Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystr NCT01648634 | Assistance Publique - Hôpitaux de Paris | Phase 3 |
| Completed | Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy NCT01521546 | Subha Raman | N/A |
| Completed | DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls NCT01523964 | Dart Therapeutics. LLC | N/A |
| Completed | Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids NCT01388764 | Massachusetts General Hospital | Phase 1 |
| Completed | Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin NCT02516085 | University Hospital, Basel, Switzerland | Phase 1 |
| Unknown | Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping NCT01385917 | Genethon | — |
| Unknown | Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystro NCT01610440 | Shenzhen Beike Bio-Technology Co., Ltd. | Phase 1 / Phase 2 |
| Completed | Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy NCT01645098 | Nationwide Children's Hospital | N/A |
| Completed | Flu Vaccine Study in Neuromuscular Patients 2011 NCT01422200 | Children's Hospital Medical Center, Cincinnati | Phase 4 |
| Completed | Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients NCT01396239 | Sarepta Therapeutics, Inc. | Phase 2 |
| Completed | Tadalafil and Sildenafil for Duchenne Muscular Dystrophy NCT01359670 | Cedars-Sinai Medical Center | EARLY_Phase 1 |
| Completed | Safety Study of Flavocoxid in Duchenne Muscular Dystrophy NCT01335295 | University of Messina | Phase 1 |
| Completed | Optimum Insufflation Capacity in NMD NCT01981915 | Universität Duisburg-Essen | N/A |
| Completed | Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy NCT01183767 | Charite University, Berlin, Germany | Phase 2 / Phase 3 |
| Completed | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) NCT01247207 | PTC Therapeutics | Phase 3 |
| Completed | Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy NCT01207908 | Children's Hospital Medical Center, Cincinnati | Phase 1 / Phase 2 |
| Terminated | Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy NCT01239758 | Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA | Phase 2 |
| Terminated | Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy NCT01168908 | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Phase 2 |
| Completed | The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parent NCT01182324 | National Human Genome Research Institute (NHGRI) | — |
| Terminated | Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy NCT01099761 | Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA | Phase 2 |
| Completed | Cardiac Magnetic Resonance in Children With Muscular Dystrophy NCT01081080 | Cooperative International Neuromuscular Research Group | — |
| Completed | This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy NCT01098708 | Washington University School of Medicine | — |
| Completed | Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies NCT01126697 | Cooperative International Neuromuscular Research Group | Phase 2 / Phase 3 |
| Terminated | Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscu NCT01009294 | PTC Therapeutics | Phase 2 |
| Completed | Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement S NCT01125709 | Cooperative International Neuromuscular Research Group | — |
| Completed | Cardiac Outcome Measures in Children With Muscular Dystrophy NCT01066455 | Cooperative International Neuromuscular Research Group | — |
| Completed | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) NCT01037309 | BioMarin Pharmaceutical | Phase 1 / Phase 2 |
| Terminated | Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders NCT00839033 | Assistance Publique - Hôpitaux de Paris | Phase 3 |
| Completed | Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy NCT00873782 | University of North Carolina, Chapel Hill | Phase 1 |
| Terminated | Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) NCT00847379 | PTC Therapeutics | Phase 2 |
| Withdrawn | Stem Cell Therapy in Duchenne Muscular Dystrophy NCT02241434 | Neurogen Brain and Spine Institute | Phase 1 |
| Completed | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) P NCT00844597 | Sarepta Therapeutics, Inc. | Phase 1 / Phase 2 |
| Unknown | Ramipril Versus Carvedilol in Duchenne and Becker Patients NCT00819845 | Catholic University, Italy | Phase 4 |
| Completed | Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) NCT00758225 | Santhera Pharmaceuticals | Phase 2 |
| Terminated | Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) NCT00759876 | PTC Therapeutics | Phase 2 |
| Completed | Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) NCT00592553 | PTC Therapeutics | Phase 2 |
| Unknown | The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy NCT00606775 | Suzuka Hospital | Phase 4 |
| Completed | Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy NCT00159250 | Imperial College London | Phase 1 / Phase 2 |
| Recruiting | The Duchenne Registry NCT02069756 | The Duchenne Registry | — |
| Terminated | CoQ10 and Prednisone in Non-Ambulatory DMD NCT00308113 | Cooperative International Neuromuscular Research Group | Phase 3 |
| Completed | Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons NCT00451074 | Nationwide Children's Hospital | Phase 1 |
| Completed | Digestive Events in Duchenne Muscular Dystrophy Patients NCT04384354 | Centre d'Investigation Clinique et Technologique 805 | — |
| Completed | Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular D NCT05281120 | Istituto Auxologico Italiano | N/A |
| Completed | Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy NCT00312247 | Shriners Hospitals for Children | — |
| Completed | Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy NCT00428935 | Nationwide Children's Hospital | Phase 1 |
| Completed | Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy NCT00264888 | PTC Therapeutics | Phase 2 |
| Unknown | Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD) NCT00468832 | Cooperative International Neuromuscular Research Group | — |
| Completed | High-dose Prednisone in Duchenne Muscular Dystrophy NCT00110669 | Cooperative International Neuromuscular Research Group | Phase 3 |
| Completed | Gentamicin Treatment of Muscular Dystrophy NCT00005574 | National Institute of Neurological Disorders and Stroke (NINDS) | Phase 1 |
| Completed | Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy NCT00004646 | National Center for Research Resources (NCRR) | Phase 3 |
| Available | Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy NCT03863119 | Santhera Pharmaceuticals | — |
| Approved For Marketing | Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy NCT02592941 | PTC Therapeutics | — |
| Available | Expanded Access Study of UC-MSC in DMD Patients NCT06579352 | MED Institute Inc. | — |
| No Longer Available | Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) NCT03433807 | Santhera Pharmaceuticals | — |
| Completed | CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers NCT01540604 | RSPR Pharma AB | Phase 2 |
| No Longer Available | Extension Study of Drisapersen in DMD Subjects NCT02636686 | BioMarin Pharmaceutical | — |