Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
NCT05232929 · View on ClinicalTrials.gov ↗
Study 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 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 This substudy was withdrawn upon implementation of protocol version 4.
Conditions Studied
Interventions
- DRUG Risdiplam
Study Locations (20)
California
- Loma Linda University Health Care — Loma Linda
- Children's Hospital Los Angeles — Los Angeles
- Valley Children's Hospital — Madera
- University California - Irvine — Orange
- University of California Davis Medical Center — Sacramento
Florida
- Nemour's Children's Hospital, Florida — Orlando
- All Children's Research Institute, Inc. — St. Petersburg
- Advent Health Orlando — Winter Park
Arizona
- Barrow Neurological Institute — Phoenix
- Phoenix Children's Hospital — Phoenix
District of Columbia
- Medstar Georgetown University Hospital — Washington D.C.
- Children's National Hospital — Washington D.C.
Kentucky
- University of Kentucky Medical Center — Lexington
- Norton Children's Hospital — Louisville
Michigan
- University of Michigan Pediatric Rehabilitation Center — Ann Arbor
- Helen DeVos Children's Hospital at Spectrum Health — Grand Rapids
Arkansas
- University of Arkansas for Medical Sciences — Little Rock
Colorado
- University of Colorado — Aurora
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 404 participants |
| Start Date | 2022-03-29 |
| Est. Completion | 2026-12-31 |
| Phase | Phase 4 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05232929
The ClinicalTrials.gov registry entry for NCT05232929 describes a study currently listed as active not recruiting. It is categorized as Phase 4, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 404 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genentech, which has 206 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 1 intervention — of which Risdiplam is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05232929 reports 20 study locations spanning 10 distinct geographic areas — top geographies include California, Florida, Arizona. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05232929 about?
NCT05232929 is a clinical study titled "Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)". 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 wi...
What is the current status of trial NCT05232929?
This trial is currently active not recruiting. It is a Phase 4 study. The enrollment target is 404 participants. The study started on 2022-03-29. Estimated completion is 2026-12-31.
What conditions does trial NCT05232929 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05232929?
The interventions under investigation include: Risdiplam (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05232929?
This trial is sponsored by Genentech, which has 206 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05232929 being conducted?
This trial has 20 study locations across Arizona, Arkansas, California, Colorado, District of Columbia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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