Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Study Status: Active Not Recruiting

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 Information (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for approximately 24 months 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. An optional sub-study was planned to assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. This substudy was withdrawn upon implementation of protocol version 4.

Sex: ALL

Researchers look for people who fit a certain description, called eligibility criteria. Below are the inclusion and exclusion criteria for study participants:

Inclusion Criteria

Provide informed consent or assent when appropriate, as determined by the participant's age and individual site and local standards
Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the risdiplam (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])

Conditions

Spinal Muscular Atrophy

Intervention/Treatment

DRUG : Risdiplam

Sponsor

Genentech, Inc.

Principal Investigator(s)

Clinical Trials, STUDY_DIRECTOR, Hoffmann-La Roche

Phase

PHASE4