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Recruiting NCT06019637

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

— Observational Spinal Muscular Atrophies

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Onasemnogene Abeparvovec.
Who it may be relevant to
Registry conditions: Spinal Muscular Atrophies. Basic parameters: 0 years — 100 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Brazil
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study

Overview

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 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

  • Other Onasemnogene Abeparvovec
    Retrospective and prospective observational study. There is no treatment allocation.

Primary outcome measures

  • Incidence and severity of treatment-emergent SAEs [Time frame: Up to 5 years]
Secondary outcome measures (5)
  • Number of patients who experience at least one AESI and number of patients by AESI [Time frame: Up to 15 years]
  • Number of patients who have survived and have not required permanent ventilatory assistance [Time frame: Up to 15 years]
  • Time until death or the need for permanent ventilatory assistance [Time frame: Up to 15 years]
  • Number of patients who achieve each Developmental Motor Milestone [Time frame: Up to 15 years]
  • Number of patients who achieve each WHO Developmental Milestone within age percentiles [Time frame: Up to 15 years]

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Brazil · 2 centers
  • Novartis Investigative Site — Curitiba
  • Novartis Investigative Site — São Paulo

Identifiers

NCT: NCT06019637 · COAV101ABR01

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗