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

Running, not enrolling · Phase 4

Conditions studied: Spinal Muscular Atrophy

In brief

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 This substudy was withdrawn upon implementation of protocol version 4.

Key facts

Study ID

NCT05232929

Run by

Genentech, Inc.

People needed

403

Starts

2022-03-29

Expected to finish

2026-12-31

Last updated by the study team

2026-07-16

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

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

Where it is running

• Barrow Neurological Institute — Phoenix, Arizona, United States
• Phoenix Children's Hospital — Phoenix, Arizona, United States
• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States
• Loma Linda University Health Care — Loma Linda, California, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Valley Children's Hospital — Madera, California, United States
• University California - Irvine — Orange, California, United States
• University of California Davis Medical Center — Sacramento, California, United States
• University of Colorado — Aurora, Colorado, United States
• Medstar Georgetown University Hospital — Washington D.C., District of Columbia, United States
• Children's National Hospital — Washington D.C., District of Columbia, United States
• Nemour's Children's Hospital, Florida — Orlando, Florida, United States
• All Children's Research Institute, Inc. — St. Petersburg, Florida, United States
• Advent Health Orlando — Winter Park, Florida, United States
• Rare Disease Research, LLC — Atlanta, Georgia, United States
• University of Kentucky Medical Center — Lexington, Kentucky, United States
• Norton Children's Hospital — Louisville, Kentucky, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• University of Michigan Pediatric Rehabilitation Center — Ann Arbor, Michigan, United States
• Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids, Michigan, United States
• Gillette Children's Specialty Healthcare — Minnetonka, Minnesota, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Washington University;Wash Uni. Sch. Of Med — St Louis, Missouri, United States
• NYU Hospital for Joint Diseases — New York, New York, United States
• Columbia University Med Center — New York, New York, United States

Full record on ClinicalTrials.gov

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