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

What this Trial Is About

This research aims to evaluate the effectiveness and safety of risdiplam in children under 2 years old diagnosed with spinal muscular atrophy (SMA) who have two SMN2 gene copies and previously received gene therapy with onasemnogene abeparvovec but have shown a plateau or decline in function. This is an open-label, single-arm, multicenter phase IV clinical study focused on pediatric patients with SMA to better understand treatment options after gene therapy. Participants will take risdiplam orally once daily for 72 weeks during the initial treatment period. Following this, there will be a 1-year treatment extension period, making the total study duration about 120 weeks (approximately 2.5 years) per participant. The risdiplam dose will be adjusted based on the child's weight and age. Throughout the study, participants will undergo assessments including the Bayley Scales of Infant and Toddler Development to measure motor skills at baseline and after 72 weeks of treatment. Researchers will monitor safety by tracking adverse and serious adverse events, as well as any treatment discontinuations due to side effects, up to 120 weeks. This study offers long-term observation to understand how risdiplam affects children with SMA post-gene therapy.

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