NCT06019637

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

A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 0 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

Detailed description

This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and treated with Onasemnogene Abeparvovec (Zolgensma®) for up to 15 years after the treatment. This study will support the benefit-risk assessment of Onasemnogene Abeparvovec in the approved indications and may also allow for detection of new safety signals and provide further guidance on the management of safety risks associated with Onasemnogene Abeparvovec to patients/caregivers, health care providers (HCPs) and treating physicians, as required by Brazilian Health Authority ANVISA as a conditional measure for granting Zolgensma®'s authorization.

Conditions

• Spinal Muscular Atrophies

Interventions

Type	Name	Description
OTHER	Onasemnogene Abeparvovec	Retrospective and prospective observational study. There is no treatment allocation.

Timeline

• Start date: 2023-11-22
• Primary completion: 2038-09-30
• Completion: 2038-09-30
• First posted: 2023-08-31
• Last updated: 2025-01-15

Locations

2 sites across 1 country: Brazil