Clinical Trial

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

Study acronym: WeSMA
Active, Not Recruiting Phase 4
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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 This substudy was withdrawn upon implementation of protocol version 4.
Trial Details
NCT Number NCT05232929
Lead Sponsor Genentech, Inc.
Conditions Spinal Muscular Atrophy
Enrollment 403 participants
Start Date 2022-03-29
Primary Completion 2026-12-31 (estimated)
Study Completion 2026-12-31 (estimated)
Updated on ClinicalTrials.gov 2026-07-16