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

  • Spinal Muscular Atrophy (SMA)
Trial Status:


This trial runs in
  • Charlottesville
  • Kansas City
  • Milwaukee
  • Orlando
  • Philadelphia
  • Phoenix
  • Salt Lake City
Trial Identifier:

NCT05232929 ML43702

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      The source of the below information is the publicly available website ClinicalTrials.gov. It has been summarised and edited into simpler language.

      The below information is taken directly from the publicly available website ClinicalTrials.gov within a week of any updates, and has not been edited.

      Results Disclaimer

      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.

      Genentech, Inc. Sponsor
      Phase 4 Phase
      NCT05232929 , ML43702 Trial Identifier
      Risdiplam Treatments
      Spinal Muscular Atrophy Condition
      Official Title

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

      Eligibility Criteria

      All Gender
      All Ages Age
      No Healthy Volunteers
      Inclusion 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)
      Exclusion Criteria
      • 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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