A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.

NCT Number	NCT05861999
Lead Sponsor	Hoffmann-La Roche
Conditions	Muscular Atrophy, Spinal
Enrollment	28 participants
Start Date	2024-08-14
Primary Completion	2028-03-31 (estimated)
Study Completion	2029-03-31 (estimated)
Updated on ClinicalTrials.gov	2026-07-09