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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
RECRUITINGChildren's Hospital of Colorado
Aurora, Colorado, United States
RECRUITINGUniversity of Florida Pediatrics
Gainesville, Florida, United States
RECRUITINGChildren's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States
RECRUITINGAnn and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, United States
NOT_YET_RECRUITINGHelen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States
RECRUITINGColumbia University Medical Center
New York, New York, United States
RECRUITINGChildren'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGThe University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States
RECRUITINGCook Children's Jane and John Justin Neurosciences Center
Fort Worth, Texas, United States
RECRUITING...and 6 more locations
Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment
The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.
Time frame: Baseline, Week 72
Percentage of Participants With Adverse Events
Time frame: Up to 120 weeks
Percentage of Participants With Serious Adverse Events
Time frame: Up to 120 weeks
Percentage of Participants With Treatment Discontinuation Due to Adverse Events
Time frame: Up to 120 weeks
Reference Study ID Number: BN44620 https://forpatients.roche.com/
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