clinical trial · NCT05861999
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
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 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.
started
Aug 14, 2024
primary completion
Mar 31, 2028
completion
Mar 31, 2029
last updated
Apr 13, 2026
official title
A Phase IV Open-Label 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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol