Actively Recruiting
Long-term Safety Study of Onasemnogene Abeparvovec (Zolgensma ) in Brazilian Patients With Spinal Muscular Atrophy (SMA) - ARISER Study
Led by Novartis Pharmaceuticals · Updated on 2025-01-15
50
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
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.
CONDITIONS
Brief Title
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Parent or legal guardian has provided signed consent.
- Genetically confirmed Spinal Muscular Atrophy with bi-allelic SMN1 mutation and clinical diagnosis of SMA Type 1 or up to 3 copies of SMN2 gene.
- Treated with Onasemnogene Abeparvovec (Zolgensma�) before enrolling.
- If previously treated with nusinersen or risdiplam before Onasemnogene Abeparvovec, currently not receiving these treatments.
- Willing and able to comply with phone contact follow-up throughout the study.
You will not qualify if you...
- Currently enrolled in any interventional clinical trial other than phase IV OFELIA trial.
- Will discontinue this study if enrolled in any pharmacological interventional clinical trial during follow-up.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - Up to 15 years
Participants are observed for long-term safety and developmental outcomes after treatment with Onasemnogene Abeparvovec (Zolgensma®).
Regular follow-up contacts by phone throughout the study
Trial Site Locations
Total: 2 locations
1
Novartis Investigative Site
Curitiba, Paraná, Brazil, 81520-060
Actively Recruiting
2
Novartis Investigative Site
São Paulo, São Paulo, Brazil, 05403-000
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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