A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Start Date
5/30/2024
Completion Date
3/31/2029
Summary
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Risdiplam
Conditions
Locations
University of Arkansas for Medical Sciences
Little Rock, Arkansas 72103
United States
Children's Hospital of Colorado
Aurora, Colorado 80045
United States
University of Florida Pediatrics
Gainesville, Florida 32610
United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia 30329-2309
United States
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois 60611
United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan 49503
United States
Columbia University Medical Center
New York, New York 10032
United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas 75390
United States
Cook Children's Jane and John Justin Neurosciences Center
Fort Worth, Texas 76104
United States
Children's Hospital of the King's Daughter
Norfolk, Virginia 23510
United States
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
Berlin, 13353
Germany
UKGM Standort Gießen
Giessen, 35392
Germany
Uniwersyteckie Centrum Kliniczne
Uniwersyteckie Centrum Kliniczne, 80-952
Poland
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730
Poland
Great Ormond Street Hospital For Children
London, WC1N 3JH
United Kingdom