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NCT05232929 · ClinicalTrials.gov registry record · Phase 4
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
A Phase 4 study of Spinal Muscular Atrophy, sponsored by Genentech.
- Active
- Registry status
- Phase 4
- Development phase
- 404
- Enrollment target
- 20
- Study locations
NCT05232929: Active Phase 4 study of Spinal Muscular Atrophy, sponsored by Genentech.
NCT05232929 is a Phase 4 study of Spinal Muscular Atrophy that is active but no longer recruiting, run by Genentech. The registered enrollment target is 404 participants, above the 91-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (344% higher). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05232929, a Phase 4 study of Spinal Muscular Atrophy, is active but no longer recruiting, sponsored by Genentech.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 4
- Development phase
- 404 participants
- Enrollment target
- 20
- Study locations
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 This substudy was withdrawn upon implementation of protocol version 4.
Primary Outcome
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Pre-existing conditions which worsen during a study a
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 |
What the registry record for NCT05232929 still lists
NCT05232929 is an interventional study that assigns participants to a tested intervention. The registered 404 participants enrollment target is mid-sized for trials with a published cap, above the 91-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (344% higher).
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.
NCT05232929 names 20 study sites across 10 states, led by California, Florida, Arizona.
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.
What interventions are being tested in trial NCT05232929?
The interventions under investigation include: Risdiplam (DRUG).
Who is sponsoring clinical trial NCT05232929?
This trial is sponsored by Genentech, which has 332 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Spinal Muscular Atrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05232929's enrollment target sits among peer trials
404 1st of 22 higher than 22 of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
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-
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
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Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
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Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
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Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
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A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
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