A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Start Date
8/14/2024
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 in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled 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
Valley Children's Hospital
Madera, California 93636
United States
Stanford Univ Medical Center
Palo Alto, California 94304
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
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan 49503
United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
University of Texas Southwestern Medical Center
Dallas, Texas 75390
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
Soroka Medical Center
Beersheba, 8410101
Israel
Schneider Children's Medical Center of Israel
Petah Tikva, 4920235
Israel
Sourasky MC, Dana-Dwek Children's Hospital
Tel Aviv, 6423906
Israel
Uniwersyteckie Centrum Kliniczne
Gda?sk, 80-952
Poland
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730
Poland
Sidra Medicine
Al Rayyan,
Qatar
Great Ormond Street Hospital For Children
London, WC1N 3JH
United Kingdom