Trials / Not Yet Recruiting
Not Yet RecruitingNCT07531719
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
A Non-lnterventional, Multi-Center Study Investigating the Outcomes of Risdiplam in Type I and Type II Spinal Muscle Atrophy Patients
- Status
- Not Yet Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 30 (estimated)
- Sponsor
- Hoffmann-La Roche · Industry
- Sex
- All
- Age
- 5 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Timeline
- Start date
- 2026-04-15
- Primary completion
- 2028-03-01
- Completion
- 2029-03-01
- First posted
- 2026-04-15
- Last updated
- 2026-04-15
Source: ClinicalTrials.gov record NCT07531719. Inclusion in this directory is not an endorsement.