A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Incidence and severity of treatment-emergent SAEs
研究概览
简要总结
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 0 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
结局指标
主要结局
Incidence and severity of treatment-emergent SAEs
时间窗: Up to 5 years
Incidence and severity of treatment-emergent Serious AEs (SAEs)
次要结局
- Number of patients who experience at least one AESI and number of patients by AESI(Up to 15 years)
- Number of patients who have survived and have not required permanent ventilatory assistance(Up to 15 years)
- Time until death or the need for permanent ventilatory assistance(Up to 15 years)
- Number of patients who achieve each Developmental Motor Milestone(Up to 15 years)
- Number of patients who achieve each WHO Developmental Milestone within age percentiles(Up to 15 years)