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

Официальное название

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

Обзор

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

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• Incidence and severity of treatment-emergent SAEs [Срок оценки: Up to 5 years]

Вторичные конечные точки (5)

• Number of patients who experience at least one AESI and number of patients by AESI [Срок оценки: Up to 15 years]
• Number of patients who have survived and have not required permanent ventilatory assistance [Срок оценки: Up to 15 years]
• Time until death or the need for permanent ventilatory assistance [Срок оценки: Up to 15 years]
• Number of patients who achieve each Developmental Motor Milestone [Срок оценки: Up to 15 years]
• Number of patients who achieve each WHO Developmental Milestone within age percentiles [Срок оценки: Up to 15 years]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Бразилия · 2 центра

• Novartis Investigative Site — Curitiba
• Novartis Investigative Site — São Paulo

Идентификаторы

NCT: NCT06019637 · COAV101ABR01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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