Study Description
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal
Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®) 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 Abeparvovec (Zolgensma®) for up to 15 years after the
treatment. This study will support the benefit-risk assessment of Onasemnogene
Abeparvovec in the approved indications and may also allow for detection of new safety
signals and provide further guidance on the management of safety risks associated with
Onasemnogene Abeparvovec to patients/caregivers, health care providers (HCPs) and
treating physicians, as required by Brazilian Health Authority ANVISA as a conditional
measure for granting Zolgensma®'s authorization.
Interventions
Onasemnogene Abeparvovec
Eligibility 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.
Worldwide Contacts
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