NCT05861986

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

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 28 (estimated)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 3 Months – 24 Months
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Muscular Atrophy, Spinal

Interventions

Type	Name	Description
DRUG	Risdiplam	Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.

Timeline

• Start date: 2024-05-30
• Primary completion: 2028-03-31
• Completion: 2029-03-31
• First posted: 2023-05-17
• Last updated: 2026-04-03

Locations

16 sites across 4 countries: United States, Germany, Poland, United Kingdom

Regulatory

• FDA-regulated drug study