Overview

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

Status:
Not yet recruiting

Trial end date:
2029-02-01

Target enrollment:
0

Participant gender:
All

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.

Phase:

Phase 4

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Genentech, Inc.

Treatments:

Risdiplam

Criteria

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])

- Aged >/=2 years with a score