Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Public ClinicalTrials.gov record NCT05232929. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 This substudy was withdrawn upon implementation of protocol version 4.
Study identification
- NCT ID
- NCT05232929
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 4
- Enrollment
- 403 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 29, 2022 (Actual)
- Primary completion
- Oct 2, 2026 (Estimated)
- Completion
- Oct 2, 2026 (Estimated)
- Last update posted
- Sep 9, 2026
2022 – 2026
United States locations
- U.S. sites
- 37
- U.S. states
- 21
- U.S. cities
- 34
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | 85013 | — |
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | — |
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72103 | — |
| Loma Linda University Health Care | Loma Linda | California | 92354 | — |
| Children's Hospital Los Angeles | Los Angeles | California | 90010 | — |
| Valley Children's Hospital | Madera | California | 93636 | — |
| University California - Irvine | Orange | California | 92868 | — |
| University of California Davis Medical Center | Sacramento | California | 95817 | — |
| University of Colorado | Aurora | Colorado | 80045 | — |
| Medstar Georgetown University Hospital | Washington D.C. | District of Columbia | 20007 | — |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | — |
| Nemour's Children's Hospital, Florida | Orlando | Florida | 32827 | — |
| All Children's Research Institute, Inc. | St. Petersburg | Florida | 33701 | — |
| Advent Health Orlando | Winter Park | Florida | 32789 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | — |
| University of Kentucky Medical Center | Lexington | Kentucky | 40536 | — |
| Norton Children's Hospital | Louisville | Kentucky | 40202 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| University of Michigan Pediatric Rehabilitation Center | Ann Arbor | Michigan | 48109 | — |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids | Michigan | 49503 | — |
| Gillette Children's Specialty Healthcare | Minnetonka | Minnesota | 55343 | — |
| Mayo Clinic | Rochester | Minnesota | 55905 | — |
| Washington University;Wash Uni. Sch. Of Med | St Louis | Missouri | 63110 | — |
| NYU Hospital for Joint Diseases | New York | New York | 10003 | — |
| Columbia University Med Center | New York | New York | 10032 | — |
| Cincinnati Childrens Hospital | Cincinnati | Ohio | 45229 | — |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | — |
| Childrens Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Texas at Austin Health sciences, Dell Medical School | Austin | Texas | 78723 | — |
| Neurology & Neuromuscular Care Center | Denton | Texas | 76208 | — |
| Central Texas Neurology Consultants | Round Rock | Texas | 78681 | — |
| Methodist Children's Hospital of South Texas | San Antonio | Texas | 78229 | — |
| University Of Utah | Salt Lake City | Utah | 84108 | — |
| University of Virginia Children?s Hospital | Charlottesville | Virginia | 22903 | — |
| MultiCare Health System Institute for Research and Innovation | Tacoma | Washington | 98405 | — |
| UBC (Remote Coordinating Center, no physical facility) | Morgantown | West Virginia | 26508 | — |
| Childrens Hospital of Wisconsin | Milwaukee | Wisconsin | 53201 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05232929, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 9, 2026 · Synced Oct 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT05232929 live on ClinicalTrials.gov.