NCT05232929

Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment

• Status: Active Not Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 404 (actual)
• Sponsor: Genentech, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Information (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for approximately 24 months from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study if they agree to continue participating in the follow-up assessments. An optional sub-study was planned to assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. This substudy was withdrawn upon implementation of protocol version 4.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Risdiplam	Risdiplam taken by participants as prescribed by their physician.

Timeline

• Start date: 2022-03-29
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2022-02-10
• Last updated: 2026-04-17

Locations

38 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study