A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
Status: Recruiting
Location: See all (16) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
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
Participation Requirements
Sex: All
Minimum Age: 3 months
Maximum Age: 2
Healthy Volunteers: f
View:
• \<2 years of age at the time of informed consent
• Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene
• Confirmed presence of two SMN2 gene copies as documented through laboratory testing
• Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
• Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to enrollment
• If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration
• Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration
Locations
United States
Arkansas
University of Arkansas for Medical Sciences
RECRUITING
Little Rock
Colorado
Children's Hospital of Colorado
RECRUITING
Aurora
Florida
University of Florida Pediatrics
RECRUITING
Gainesville
Georgia
Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITING
Atlanta
Illinois
Ann and Robert H. Lurie Children Hospital of Chicago
NOT_YET_RECRUITING
Chicago
Michigan
Helen DeVos Children's Hospital at Spectrum Health
RECRUITING
Grand Rapids
New York
Columbia University Medical Center
RECRUITING
New York
Pennsylvania
Children'S Hospital of Philadelphia
RECRUITING
Philadelphia
Texas
The University of Texas Southwestern Medical Center at Dallas
RECRUITING
Dallas
Cook Children's Jane and John Justin Neurosciences Center
RECRUITING
Fort Worth
Virginia
Children's Hospital of the King's Daughter
RECRUITING
Norfolk
Other Locations
Germany
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
RECRUITING
Berlin
UKGM Standort Gießen
RECRUITING
Giessen
Poland
Uniwersyteckie Centrum Kliniczne
RECRUITING
Gda?sk
Instytut Pomnik Centrum Zdrowia Dziecka
RECRUITING
Warsaw
United Kingdom
Great Ormond Street Hospital For Children
RECRUITING
London
Contact Information
Primary
Reference Study ID Number: BN44620 https://forpatients.roche.com/
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. Only)
Time Frame
Start Date:2024-05-30
Estimated Completion Date:2029-03-31
Participants
Target number of participants:28
Treatments
Experimental: Risdiplam
Participants will receive risdiplam orally once daily for 72 weeks (Treatment Period). The Treatment Period will be followed by a 1-year Treatment Extension Period for a total study duration of 120 weeks (approximately 2.5 years) for each participant enrolled.