Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (NCT05232929)
Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
🩺 Plain-English Summary (8th-Grade Level)
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 This substudy was withdrawn upon implementation of protocol version 4.
Who can join: Review the eligibility criteria below.
Location: Barrow Neurological Institute — Phoenix, Arizona
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (38 Sites)
Showing the first 25 of 38 sites.
- Barrow Neurological Institute — Phoenix, Arizona
- Phoenix Children's Hospital — Phoenix, Arizona
- University of Arkansas for Medical Sciences — Little Rock, Arkansas
- Loma Linda University Health Care — Loma Linda, California
- Children's Hospital Los Angeles — Los Angeles, California
- Valley Children's Hospital — Madera, California
- University California - Irvine — Orange, California
- University of California Davis Medical Center — Sacramento, California
- University of Colorado — Aurora, Colorado
- Medstar Georgetown University Hospital — Washington D.C., District of Columbia
- Children's National Hospital — Washington D.C., District of Columbia
- Nemour's Children's Hospital, Florida — Orlando, Florida
- All Children's Research Institute, Inc. — St. Petersburg, Florida
- Advent Health Orlando — Winter Park, Florida
- Rare Disease Research, LLC — Atlanta, Georgia
- University of Kentucky Medical Center — Lexington, Kentucky
- Norton Children's Hospital — Louisville, Kentucky
- Massachusetts General Hospital — Boston, Massachusetts
- University of Michigan Pediatric Rehabilitation Center — Ann Arbor, Michigan
- Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids, Michigan
- Gillette Children's Specialty Healthcare — Minnetonka, Minnesota
- Mayo Clinic — Rochester, Minnesota
- Washington University;Wash Uni. Sch. Of Med — St Louis, Missouri
- NYU Hospital for Joint Diseases — New York, New York
- Columbia University Med Center — New York, New York
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Spinal Muscular Atrophy
- Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb — Pittsburgh, Pennsylvania
- A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) — Phoenix, Arizona
- Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab — Phoenix, Arizona
- Spinraza in Adult Spinal Muscular Atrophy — Phoenix, Arizona
- Early Check: Expanded Screening in Newborns — Research Triangle Park, North Carolina
Explore by condition: All Spinal Muscular Atrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.