Long-term safety evaluation of Zolgensma in Brazilian SMA patients
Official title: A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study
Run by Novartis · NCT06019637 · Data updated 2026-11-12
This study is looking at the long-term safety of the Zolgensma treatment in Brazilian children with Spinal Muscular Atrophy to see how it affects them over time.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 0 Years to 100 Years |
| Sex | All |
| Enrollment | 50 (estimated) |
| Sponsor | Novartis Industry-sponsored |
| Locations | 2 sites (Curitiba, Paraná and 1 other locations) |
| Status | Recruiting |
| Started | November 27, 2023 |
| Main results expected | January 2, 2042 |
| Study ends | January 2, 2042 |
| Registry record updated | November 10, 2026 |
| Trial ID | NCT06019637 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include Brazilian pediatric patients with a confirmed diagnosis of SMA who have been treated with Zolgensma.
✕Probably not a fit
Patients currently enrolled in any interventional clinical trial other than the phase IV OFELIA trial may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study aims to gather long-term safety data on Brazilian pediatric patients diagnosed with Spinal Muscular Atrophy (SMA) who have been treated with Onasemnogene Abeparvovec (Zolgensma®). It is a non-interventional Post Authorization Safety Study (PASS) that will monitor these patients for up to 15 years post-treatment. The study is designed to support the benefit-risk assessment of Zolgensma and may help identify new safety signals, providing valuable information for patients, caregivers, and healthcare providers. The data collected will also assist in managing safety risks associated with the treatment as mandated by the Brazilian Health Authority, ANVISA.
Why it matters
Potential benefit: If successful, this study could enhance the understanding of the long-term safety profile of Zolgensma, leading to improved patient management and outcomes.
How similar studies have gone: While this study focuses on long-term safety data, similar observational studies have shown success in monitoring treatment effects and safety in other conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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.
Where it's running
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Novartis Investigative Site
Curitiba, Paraná, Brazil Recruiting -
Novartis Investigative Site
São Paulo, São Paulo, Brazil Recruiting
Study contacts
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: +41613241111
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.