NCT06019637Recruiting
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
Source: ClinicalTrials.gov · Last updated 15 Jan 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 50 (estimated)
- First submitted
- 25 Aug 2023
- Start date
- 22 Nov 2023
- Primary completion date
- 30 Sept 2038
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT06019637
What you commit to
- Study type: Observational
- Planned participants: 50
- From 22 Nov 2023 to the primary completion date, 30 Sept 2038
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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.
Who the study is looking for
- 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
Age in the registry: 0 years to 100 years · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Curitiba | Brazil | Recruiting |
| - Coordinator of this site? Confirm its status | São Paulo | Brazil | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.