Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
NCT ID: NCT05232929
Last Updated: 2026-01-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE4
404 participants
INTERVENTIONAL
2022-03-29
2026-12-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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 This substudy was withdrawn upon implementation of protocol version 4.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
NCT04256265
Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study
NCT05839145
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
NCT07265232
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
NCT05337553
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
NCT07070999
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Risdiplam
Participants who are taking risdiplam prescribed based on clinician judgment, as per the Evrysdi® USPI are enrolled in this study.
A Remote Virtual Site option is available in the study allowing enrollment of participants with SMA who are prescribed risdiplam treatment even if their physician is not a participating investigator. A central investigator will be responsible for data collection and data entry for participants who do not have a participating investigator.
Risdiplam
Risdiplam taken by participants as prescribed by their physician.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Risdiplam
Risdiplam taken by participants as prescribed by their physician.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the risdiplam (Evrysdi) USPI, after U.S. FDA approval (07 August 2020)
Exclusion Criteria
* Participated in a registrational trial for risdiplam (i.e., Firefish \[NCT02913482\], Sunfish \[NCT02908685\], Jewelfish \[NCT03032172\], and Rainbowfish \[NCT03779334\])
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Genentech, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
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
Cincinnati Childrens Hospital
Cincinnati, Ohio, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas at Austin Health sciences, Dell Medical School
Austin, Texas, United States
Neurology & Neuromuscular Care Center
Denton, Texas, United States
Central Texas Neurology Consultants
Round Rock, Texas, United States
Methodist Children's Hospital of South Texas
San Antonio, Texas, United States
University Of Utah
Salt Lake City, Utah, United States
University of Virginia Children?s Hospital
Charlottesville, Virginia, United States
MultiCare Health System Institute for Research and Innovation
Tacoma, Washington, United States
UBC (Remote Coordinating Center, no physical facility)
Morgantown, West Virginia, United States
Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Instituto de Rehabilitación del Caribe
Santurce, , Puerto Rico
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
ML43702
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.