Phase IV Study of Risdiplam as Early Treatment for Children Under 2 Years with Spinal Muscular Atrophy After Gene Therapy

What this Trial Is About

Researchers are evaluating the effectiveness and safety of risdiplam as an early treatment in young children under 2 years old who have spinal muscular atrophy SMA with 2 SMN2 gene copies and who have previously received onasemnogene abeparvovec gene therapy. This open-label, phase IV study focuses on pediatric participants with genetically confirmed SMA to understand how risdiplam works following gene therapy. Participants will receive risdiplam orally once daily for 72 weeks during the Treatment Period. After this, they may enter a 1-year Treatment Extension Period, making the total study duration about 120 weeks approximately 2.5 years. The dose of risdiplam will be adjusted based on the childs weight and age to suit their needs. Throughout the study, children will be monitored regularly to assess their motor development using the Bayley Scales of Infant and Toddler Development, Third Edition BSID-III Gross Motor Score. Researchers will also track adverse events, serious adverse events, and any treatment discontinuations related to side effects for up to 120 weeks. This comprehensive monitoring helps evaluate both the benefits and safety of risdiplam in this population.

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy