Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
- Muscle And Peripheral Nerve Disease
- Spinal Muscular Atrophy (SMA)
Recruiting
- United States
NCT05232929 ML43702
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.
Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
Eligibility Criteria
- Confirmed diagnosis of 5q-autosomal recessive SMA
- Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the Evrysdi® USPI, after U.S. FDA approval (07 August 2020)
- Hypersensitivity to risdiplam
- Participated in a registrational trial for risdiplam (i.e., Firefish [NCT02913482], Sunfish [NCT02908685], Jewelfish [NCT03032172], and Rainbowfish [NCT03779334])
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