Long-Term Safety Study of Zolgensma in Brazilian Children with Spinal Muscular Atrophy

What this Trial Is About

Researchers are conducting a long-term safety study to observe Brazilian pediatric patients diagnosed with Spinal Muscular Atrophy SMA who have been treated with Onasemnogene Abeparvovec Zolgensma. This observational study collects real-world safety data for up to 15 years after treatment, supporting ongoing benefit-risk assessment and monitoring for new safety signals as required by Brazilian Health Authority ANVISA. The study involves pediatric patients with SMA who have already been treated with Onasemnogene Abeparvovec. It is non-interventional, meaning patients continue their usual care while researchers observe and collect data. Participants may be enrolled on the day of treatment or retrospectively if prior medical history is available. No new treatments are assigned, and there is no control or placebo group. Participants and their caregivers will be followed for up to 15 years through phone contacts and medical assessments to monitor safety events, survival, ventilatory support needs, and developmental milestones. Researchers will evaluate treatment-emergent serious adverse events within the first 5 years and track several secondary outcomes including adverse events of special interest, survival without permanent ventilatory assistance, and achievement of motor and developmental milestones over the full 15-year period.

A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma