Actively Recruiting
Long-term Safety Study of Onasemnogene Abeparvovec (Zolgensma) in Brazilian Patients With Confirmed Spinal Muscular Atrophy ARISER Observational Study
Led by Novartis Pharmaceuticals · Updated on 2025-01-15
50
Participants Needed
2
Research Sites
775 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the long-term safety of Onasemnogene Abeparvovec (Zolgensma4) in Brazilian pediatric patients diagnosed with Spinal Muscular Atrophy (SMA). This non-interventional Post Authorization Safety Study (PASS) aims to collect real-world safety data for up to 15 years after treatment. The study supports the benefit-risk assessment of Zolgensma4 and helps detect new safety signals, providing guidance on managing safety risks to patients, caregivers, healthcare providers, and treating physicians as required by the Brazilian Health Authority ANVISA. This observational study involves patients who have already been treated with Onasemnogene Abeparvovec (Zolgensma4). There is no treatment allocation or intervention by the study team. Patients previously treated with nusinersen or risdiplam may be included if they are not currently receiving those treatments. Participants can enroll on the day of treatment or retrospectively if medical history is available, following local ethical requirements. Participants and their parents or guardians will be contacted by phone throughout the study to collect safety information. Researchers will monitor the incidence and severity of serious adverse events related to treatment for up to 5 years. The study may continue for up to 15 years to gather long-term safety data. This ongoing monitoring helps ensure the safety of patients treated with Onasemnogene Abeparvovec (Zolgensma4).
CONDITIONS
Official 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...
- Subject's parent or legal guardian has provided signed informed consent form.
- Subject has genetically confirmed Spinal Muscular Atrophy with bi-allelic mutation in SMN1 gene and clinical diagnosis of SMA Type 1 or up to 3 copies of SMN2 gene.
- Subject has been treated with Onasemnogene Abeparvovec (Zolgensma4) prior to enrolling.
- Subjects previously treated with nusinersen or risdiplam can be enrolled if not currently receiving those treatments.
- Subject and parent/guardian are willing and able to comply with phone contacts during the study.
You will not qualify if you...
- Patients currently enrolled in any interventional clinical trial other than the phase IV OFELIA trial are excluded.
- Subjects previously enrolled in any clinical trial but not currently enrolled may be included.
- Subjects who enroll in a clinical trial with pharmacological intervention during follow-up will discontinue from this study.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here