Overview

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

Status:
Not yet recruiting

Trial end date:
2028-03-31

Target enrollment:

Participant gender:

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.

Phase:

Phase 4

Details

Lead Sponsor:

Hoffmann-La Roche

Treatments:

Risdiplam