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 Muscular Atrophy, Spinal 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
  • University of Colorado; Anschutz Medical Campus Department of Neurology accepting new patients
    Aurora Colorado 80045 United States

Details

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