Summary

Location
at UC Davis UC Irvine
Dates
study started
completion around

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 SMA. In this study, participants will be followed for the duration of the study 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…

  • Clinical diagnosis of 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 California Davis Medical Center accepting new patients
    Sacramento California 95817 United States
  • Children's Hospital Los Angeles accepting new patients
    Los Angeles California 90010 United States
  • Valley Children's Hospital accepting new patients
    Madera California 93636 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