Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Phase 4 – monitoring after market approval
Conditions:
Spinal Muscular Atrophy
Sponsor: Genentech, Inc.
trial.available_in:
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Overview
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 This substudy was withdrawn upon implementation of protocol version 4.
Who can participate
Inclusion Criteria:
* Provide informed consent or assent when appropriate, as determined by the participant's age and individual site and local standards
* 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:
* Hypersensitivity to risdiplam
* Participated in a registrational trial for risdiplam (i.e., Firefish \[NCT02913482\], Sunfish \[NCT02908685\], Jewelfish \[NCT03032172\], and Rainbowfish \[NCT03779334\])
Interventions
Risdiplam
DRUG
Locations
38
Puerto Rico (1)
Instituto de Rehabilitación del Caribe
Santurce
United States (37)
University of Michigan Pediatric Rehabilitation Center
Ann Arbor , Michigan
Rare Disease Research, LLC
Atlanta , Georgia
University of Colorado
Aurora , Colorado
University of Texas at Austin Health sciences, Dell Medical School
Austin , Texas
Massachusetts General Hospital
Boston , Massachusetts
University of Virginia Children?s Hospital
Charlottesville , Virginia
Cincinnati Childrens Hospital
Cincinnati , Ohio
Neurology & Neuromuscular Care Center
Denton , Texas
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids , Michigan
Penn State Milton S. Hershey Medical Center
Hershey , Pennsylvania
University of Kentucky Medical Center
Lexington , Kentucky
University of Arkansas for Medical Sciences
Little Rock , Arkansas
Loma Linda University Health Care
Loma Linda , California
Children's Hospital Los Angeles
Los Angeles , California
Norton Children's Hospital
Louisville , Kentucky
Valley Children's Hospital
Madera , California
Childrens Hospital of Wisconsin
Milwaukee , Wisconsin
Gillette Children's Specialty Healthcare
Minnetonka , Minnesota
UBC (Remote Coordinating Center, no physical facility)
Morgantown , West Virginia
NYU Hospital for Joint Diseases
New York , New York
Columbia University Med Center
New York , New York
University California - Irvine
Orange , California
Nemour's Children's Hospital, Florida
Orlando , Florida
Childrens Hospital of Philadelphia
Philadelphia , Pennsylvania
Barrow Neurological Institute
Phoenix , Arizona
Phoenix Children's Hospital
Phoenix , Arizona
Mayo Clinic
Rochester , Minnesota
Central Texas Neurology Consultants
Round Rock , Texas
University of California Davis Medical Center
Sacramento , California
University Of Utah
Salt Lake City , Utah
Methodist Children's Hospital of South Texas
San Antonio , Texas
Washington University;Wash Uni. Sch. Of Med
St Louis , Missouri
All Children's Research Institute, Inc.
St. Petersburg , Florida
MultiCare Health System Institute for Research and Innovation
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