NCT05861999

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

A Phase IV Open-Label 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

• 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 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.

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: 2024-08-14
• Primary completion: 2028-03-31
• Completion: 2029-03-31
• First posted: 2023-05-17
• Last updated: 2026-04-13

Locations

19 sites across 6 countries: United States, Germany, Israel, Poland, Qatar, United Kingdom

Regulatory

• FDA-regulated drug study