Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- Biogen
- 入组人数
- 200
- 试验地点
- 12
- 主要终点
- Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants
研究概览
简要总结
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed 5q-SMA.
排除标准
- •Other types of SMA (non 5q-SMA).
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
研究组 & 干预措施
DMT Treated Participants
Participants with SMA who received prior treatment with DMTs including nusinersen will be followed prospectively for up to 60 months and the available data is collected retrospectively.
Untreated Participants
Participants with SMA who received no treatment will be followed prospectively for up to 60 months.
结局指标
主要结局
Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants
时间窗: Up to 60 Months
The natural history characteristics will include the categories of the evolution of the patient's condition over the time of data collection from the registry, to describe the natural history of the disease in a real-life situation.
Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants
时间窗: Up to 60 Months
The DMT utilization characteristics will include the categories of the evolution of the DMT treated patient's condition over the time of data collection from the registry, to describe the utilization of DMTs of adult SMA patients in a real-life situation.
次要结局
未报告次要终点