Status:
RECRUITING
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Spinal Muscular Atrophies
Eligibility:
All Genders
Up to 100 years
Brief Summary
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Detailed Description
This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and treated with Onasemnogene...
Eligibility Criteria
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.
Key Trial Info
Start Date :
November 22 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 30 2038
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT06019637
Start Date
November 22 2023
End Date
September 30 2038
Last Update
January 15 2025
Active Locations (2)
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1
Novartis Investigative Site
Curitiba, Paraná, Brazil, 81520-060
2
Novartis Investigative Site
São Paulo, São Paulo, Brazil, 05403-000