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

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.

NCT Number	NCT05861986
Lead Sponsor	Hoffmann-La Roche
Conditions	Muscular Atrophy, Spinal
Enrollment	28 participants
Start Date	2024-05-30
Primary Completion	2028-03-31 (estimated)
Study Completion	2029-03-31 (estimated)
Updated on ClinicalTrials.gov	2026-07-06