Phase IV Study of Daily Oral Risdiplam in Children Under 2 Years With Spinal Muscular Atrophy Who Have Had Gene Therapy and Showed No Improvement or Decline

What this Trial Is About

This research aims to evaluate the effectiveness and safety of risdiplam in children under 2 years of age with spinal muscular atrophy SMA who have two SMN2 gene copies and have previously received gene therapy with onasemnogene abeparvovec but now show a plateau or decline in their function. The study focuses on pediatric participants diagnosed genetically with SMA and experiencing these specific issues after gene therapy. Participants will receive risdiplam orally once daily for a treatment period of 72 weeks, with the dose adjusted according to weight and age. Following this treatment period, there will be a 1-year extension phase, making the total study duration approximately 120 weeks about 2.5 years for each participant. During the study, participants will be regularly assessed, including measuring changes from baseline in motor development using the Bayley Scales of Infant and Toddler Development at 72 weeks. Researchers will monitor adverse events and treatment discontinuation throughout the entire 120 weeks. The study involves close safety monitoring and evaluation of motor function changes, with ongoing assessments during both the treatment and extension periods.

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