NCT06138639 RECRUITING A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Solid Biosciences Inc.
NCT03368742 ACTIVE NOT RECRUITING Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Solid Biosciences Inc.
NCT06450639 RECRUITING A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
Hoffmann-La Roche
NCT07429240 NOT YET RECRUITING PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Precision BioSciences, Inc.
NCT07038824 RECRUITING 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.
NCT07037862 RECRUITING 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.
NCT07287189 RECRUITING Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Satellos Bioscience, Inc.
NCT06128564 ACTIVE NOT RECRUITING A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
Hoffmann-La Roche
NCT06280209 RECRUITING A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
BioMarin Pharmaceutical
NCT03340675 ACTIVE NOT RECRUITING Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Cumberland Pharmaceuticals
NCT04906460 RECRUITING Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Wave Life Sciences Ltd.
NCT07058662 RECRUITING A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
Belief BioMed (Beijing) Co., Ltd
NCT05996003 RECRUITING NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
NS Pharma, Inc.
NCT05540860 ACTIVE NOT RECRUITING A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
Edgewise Therapeutics, Inc.
NCT06100887 ACTIVE NOT RECRUITING Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
Edgewise Therapeutics, Inc.
NCT04428476 ACTIVE NOT RECRUITING Open-label Extension of the HOPE-2 Trial
Capricor Inc.
NCT07209332 NOT YET RECRUITING Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Wave Life Sciences Ltd.
NCT04433234 ACTIVE NOT RECRUITING Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Daiichi Sankyo Co., Ltd.
NCT06867107 ENROLLING BY INVITATION A Long-term Follow-up Study for Participants That Completed the SAT-3247-CL-101 Study
Satellos Bioscience, Inc.
NCT06053814 RECRUITING NS-050/NCNP-03 in Boys With DMD (Meteor50)
NS Pharma, Inc.
NCT06643442 NOT YET RECRUITING Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
Sebastiano Lava
NCT05524883 ACTIVE NOT RECRUITING Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Dyne Therapeutics
NCT05135663 ACTIVE NOT RECRUITING Extension Study of NS-089/NCNP-02 in DMD
Nippon Shinyaku Co., Ltd.
NCT06769633 RECRUITING Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
Italfarmaco
NCT06290713 RECRUITING Vasodilator and Exercise Study for DMD (VASO-REx)
University of Florida
NCT06244082 ACTIVE NOT RECRUITING Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
Avidity Biosciences, Inc.
NCT04322357 ACTIVE NOT RECRUITING Twice Weekly Steroids and Exercise as Therapy for DMD
University of Florida
NCT05753462 RECRUITING Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy
Sqy Therapeutics
NCT06328725 NOT YET RECRUITING Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
ENCell
NCT06079736 TERMINATED A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
PepGen Inc
NCT05185622 COMPLETED A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
Santhera Pharmaceuticals
NCT04240314 COMPLETED AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Megan Waldrop
NCT05670730 COMPLETED Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
Avidity Biosciences, Inc.
NCT06833931 WITHDRAWN A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
PepGen Inc
NCT03333590 COMPLETED Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
Kevin Flanigan
NCT04004065 TERMINATED Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Sarepta Therapeutics, Inc.
NCT05938023 TERMINATED A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy
Percheron Therapeutics
NCT02485938 COMPLETED HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Capricor Inc.
NCT03375164 COMPLETED A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Sarepta Therapeutics, Inc.
NCT02606136 TERMINATED Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Kyntra Bio
NCT04956289 COMPLETED Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
NS Pharma, Inc.
NCT06304064 COMPLETED Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)
Capricor Inc.
NCT04336826 COMPLETED A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
PTC Therapeutics
NCT02667483 COMPLETED Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Daiichi Sankyo Co., Ltd.
NCT04179409 COMPLETED A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
Kevin Flanigan
NCT02354781 COMPLETED Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
Jerry R. Mendell
NCT03985878 TERMINATED A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
Sarepta Therapeutics, Inc.
NCT03439670 COMPLETED A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
ReveraGen BioPharma, Inc.
NCT03558958 TERMINATED Safety and Efficacy of P-188 NF in DMD Patients
Phrixus Pharmaceuticals, Inc.
NCT03167255 COMPLETED Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
NS Pharma, Inc.
NCT04129294 COMPLETED Exploratory Study of NS-089/NCNP-02 in DMD
National Center of Neurology and Psychiatry, Japan
NCT03796637 COMPLETED A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
PTC Therapeutics
NCT03648827 COMPLETED A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
PTC Therapeutics
NCT02964377 COMPLETED Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
Craig McDonald, MD
NCT03218995 COMPLETED Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
Sarepta Therapeutics, Inc.
NCT02740972 COMPLETED Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
NS Pharma, Inc.
NCT03038399 COMPLETED Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
ReveraGen BioPharma, Inc.
NCT02420379 COMPLETED Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc.
NCT02907619 TERMINATED An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
Pfizer
NCT02515669 TERMINATED Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
Hoffmann-La Roche
NCT02310906 COMPLETED Phase I/II Study of SRP-4053 in DMD Patients
Sarepta Therapeutics, Inc.
NCT02819557 COMPLETED Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
PTC Therapeutics
NCT02752048 COMPLETED A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
Taiho Pharmaceutical Co., Ltd.
NCT02858362 TERMINATED Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
Summit Therapeutics
NCT02760277 COMPLETED An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
ReveraGen BioPharma, Inc.
NCT02525302 TERMINATED HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
Akashi Therapeutics
NCT02760264 COMPLETED A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
ReveraGen BioPharma, Inc.
NCT02704325 WITHDRAWN Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
Kevin Flanigan
NCT02958202 TERMINATED Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
BioMarin Pharmaceutical