Long-term safety of Zolgensma in Brazilian patients with spinal muscular atrophy
Follows Brazilian people with spinal muscular atrophy treated with Zolgensma for long-term safety.
Plain-language title generated from the registry record
Registry title: A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Key facts
- Where
- Curitiba and São Paulo, BrazilSee the 2 sites
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 0 and 100 years old?
Source: NCT06019637, who can take part
Where in Brazil
- CuritibaCuritiba, BrazilTaking people
- São PauloSão Paulo, BrazilTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharmaceuticals
- Trial code
- NCT06019637
- Record updated
- 10 November 2026
- Original description
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)Read full original descriptionShow less
A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Inclusion: Subject's parent or legal guardian has provided signed eICF; Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene; Subject treated* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this study; Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec (Zolgensma®) can be enrolled if currently not receiving it; *Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec (Zolgensma®) or if prior medical history is available to complete all assessments retrospectively, in accordance with local ethical requirements; Subject and parent/guardian are willing and able to comply with the phone contacts through the course of the study.
Exclusion: Patients currently enrolled in any interventional clinical trial** other than the phase IV OFELIA trial will be excluded from the study; Subjects who were enrolled in a clinical trial (independently of the disease indication and interventional treatment) but are not currently enrolled, can be included in this study; During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.