A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Study Identifier:
BN44620
ClinicalTrials.gov Identifier:
NCT05861986
EudraCT Identifier:
N/A
Sponsor:
Hoffmann-La Roche
Recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: risdiplam
Date
May 2024 - Jan 2027
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 3 - 24 Months
Requirements Information

Protocol 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.

Trial Locations

Location
Status
Location
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72103
Status
Recruiting
Location
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
Status
Recruiting
Location
University of Florida Pediatrics
Gainesville, Florida, United States, 32610
Status
Recruiting
Location
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States, 30329-2309
Status
Recruiting
Location
Ann and Robert H. Lurie Children Hospital of Chicago
Chicago, Illinois, United States, 60611
Status
Not yet recruiting
Location
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States, 49503
Status
Recruiting

Contact Cure SMA

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