Phase 1 Muscular Dystrophy Clinical Trials
15 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–15 of 15 trials
Recruiting
Phase 1Phase 2
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Precision BioSciences, Inc.18 enrolled1 locationNCT07429240
Recruiting
Phase 1
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Insmed Gene Therapy LLC12 enrolled10 locationsNCT06817382
Recruiting
Phase 1Phase 2
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
BioMarin Pharmaceutical18 enrolled8 locationsNCT06280209
Recruiting
Phase 1
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Muscular Dystrophy, Duchenne
Sarepta Therapeutics, Inc.83 enrolled7 locationsNCT04626674
Recruiting
Phase 1Phase 2
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Duchenne Muscular Dystrophy (DMD)
Entrada Therapeutics, Inc.24 enrolled15 locationsNCT07038824
Recruiting
Phase 1Phase 2
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
Duchenne Muscular Dystrophy (DMD)
Entrada Therapeutics, Inc.24 enrolled14 locationsNCT07037862
Recruiting
Phase 1
Trial of Cell Based Therapy for DMD
Duchenne Muscular Dystrophy
Masonic Cancer Center, University of Minnesota8 enrolled1 locationNCT06692426
Recruiting
Phase 1Phase 2
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Duchenne Muscular Dystrophy
Solid Biosciences Inc.60 enrolled15 locationsNCT06138639
Recruiting
Phase 1
Clenbuterol to Target DUX4 in FSHD
Muscular Dystrophy, Facioscapulohumeral
Jeffrey Statland30 enrolled3 locationsNCT06721299
Recruiting
Phase 1Phase 2
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Muscular DystrophyLimb Girdle Muscular DystrophyLGMD2I+6 more
AskBio Inc10 enrolled6 locationsNCT05230459
Recruiting
Phase 1
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
Duchenne Muscular Dystrophy (DMD)
Grünenthal GmbH24 enrolled1 locationNCT07347548
Recruiting
Phase 1Phase 2
A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia
Oculopharyngeal Muscular Dystrophy
Benitec Biopharma, Inc.30 enrolled1 locationNCT06185673
Recruiting
Phase 1Phase 2
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Duchenne Muscular Dystrophy
Wave Life Sciences Ltd.26 enrolled5 locationsNCT04906460
Recruiting
Phase 1
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD - Facioscapulohumeral Muscular Dystrophy
Restem, LLC.16 enrolled1 locationNCT07086521
Recruiting
Phase 1Phase 2
NS-050/NCNP-03 in Boys With DMD (Meteor50)
Duchenne Muscular Dystrophy
NS Pharma, Inc.20 enrolled18 locationsNCT06053814