Phase 2 Spinal Muscular Atrophy (SMA) Clinical Trials

2 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 1–2 of 2 trials

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 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