Summary

Location
at Orange, California and other locations
Dates
study started
estimated completion

Description

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.

Official Title

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

Keywords

Spinal Muscular Atrophy, Muscular Atrophy, Atrophy, Risdiplam

Eligibility

You can join if…

  • 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)

You CAN'T join if...

  • Hypersensitivity to risdiplam
  • Participated in a registrational trial for risdiplam (i.e., Firefish [NCT02913482], Sunfish [NCT02908685], Jewelfish [NCT03032172], and Rainbowfish [NCT03779334])

Locations

  • University California - Irvine accepting new patients
    Orange California 92868 United States
  • Phoenix Children's Hospital accepting new patients
    Phoenix Arizona 85016 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Genentech, Inc.
ID
NCT05232929
Phase
Phase 4 research study
Study Type
Interventional
Participants
Expecting 500 study participants
Last Updated