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Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Sponsor: Genentech, Inc.
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 This substudy was withdrawn upon implementation of protocol version 4.
Official title: Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
404
Start Date
2022-03-29
Completion Date
2026-12-31
Last Updated
2026-01-16
Healthy Volunteers
No
Conditions
Interventions
Risdiplam
Risdiplam taken by participants as prescribed by their physician.
Locations (38)
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