Phase 1 Muscular Atrophy Clinical Trials
7 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 1–7 of 7 trials
Recruiting
Phase 1
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
Muscular Atrophy, Spinal
Biogen58 enrolled19 locationsNCT06555419
Recruiting
Phase 1Phase 2
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
Spinal Muscular Atrophy Type I
Gemma Biotherapeutics22 enrolled1 locationNCT07070999
Recruiting
Phase 1Phase 2
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
Spinal Muscular Atrophy
Lantu Biopharma20 enrolled1 locationNCT06288230
Recruiting
Phase 1Phase 2
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
Spinal Muscular Atrophy Type 3
GeneCradle Inc21 enrolled1 locationNCT06421831
Recruiting
Phase 1Phase 2
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
Spinal Muscular Atrophy
GeneCradle Inc18 enrolled4 locationsNCT05824169
Recruiting
Phase 1Phase 2
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
Spinal Muscular Atrophy (SMA)
Biocad40 enrolled7 locationsNCT05747261
Completed
Phase 1
A Phase 1 trial assessing the Safety, Tolerability, and Pharmacokinetics of NIDO-361 in Healthy Male Subjects
Spinal and bulbar muscular atrophy (SBMA)
Nido Biosciences, Inc.64 enrolled1 locationACTRN12622000964718