A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-11-22

Study Completion Date

2038-09-30

Brief Summary

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A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

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Detailed Description

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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.

Conditions

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Spinal Muscular Atrophies

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Spinal Muscular Atrophy Patients

Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec

Onasemnogene Abeparvovec

Intervention Type OTHER

Retrospective and prospective observational study. There is no treatment allocation.

Interventions

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Onasemnogene Abeparvovec

Retrospective and prospective observational study. There is no treatment allocation.

Intervention Type OTHER

Other Intervention Names

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Zolgensma

Eligibility Criteria

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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.

Minimum Eligible Age

0 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No