Phase 2 Spinal Muscular Atrophy Clinical Trials
8 recruitingPhase 2
What is a Phase 2 trial?
Phase 2 trials evaluate whether a treatment works for a specific condition and further assess its safety. These studies typically enroll several hundred participants and provide preliminary data on effectiveness.
Showing 1–8 of 8 trials
Recruiting
Phase 2
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Spinal Muscular AtrophySMASpinal Muscular Atrophy Type 3+3 more
Scholar Rock, Inc.52 enrolled25 locationsNCT07047144
Recruiting
Phase 2
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA)
argenx60 enrolled17 locationsNCT07287982
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 2
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
Spinal Muscular Atrophy
NMD Pharma A/S54 enrolled31 locationsNCT05794139
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