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Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — WeSMA  

Spinal Muscular Atrophy Clinical Trial

Official title:
Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment

Clinical Trial 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 Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05232929
Study type Interventional
Source Genentech, Inc.
Contact Reference Study ID Number: ML43702 https://forpatients.roche.com
Phone 888-662-6728 (U.S. Only)
Email global-roche-genentech-trials@gene.com
Status Recruiting
Phase Phase 4
Start date June 14, 2022
Completion date June 1, 2029
View Details
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