clinical trial · NCT05861986
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Hoffmann-La Roche·phase4·recruiting·n = 28
Muscular Atrophy, SpinalRisdiplam
brief 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.
started
May 30, 2024
primary completion
Mar 31, 2028
completion
Mar 31, 2029
last updated
Apr 3, 2026
official title
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol