Clinical Trial

A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)

Recruiting
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Summary
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with Type I and Type II Spinal Muscle Atrophy over 24 months of treatment. The study will allow retrospective collection of medical history data and previous participants visits from the medical records.
Protocol Amendment History 1 change
critical Recruitment opened 2026-08-05
Trial Details
NCT Number NCT07531719
Lead Sponsor Hoffmann-La Roche
Conditions Spinal Muscle Atrophy
Enrollment 30 participants
Start Date 2026-07-22
Primary Completion 2028-03-01 (estimated)
Study Completion 2029-03-01 (estimated)
Updated on ClinicalTrials.gov 2026-08-04