Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Change in upper limb motor function
研究概览
简要总结
Observational study of adult patients with spinal muscular atrophy types 2 and 3 receiving nusinersen
详细描述
Observational study to assess effects of nusinersen on motor function in adult patients with spinal muscular atrophy who are both ambulatory and non-ambulatory. Subjects will receive standard of care with nusinersen intrathecal injection and undergo baseline and every 6 month motor assessments and pulmonary function testing during the first two years of treatment with nusinersen.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed 5q SMA
- •ability to access intrathecal space for nusinersen injection
排除标准
- •Renal impairment
- •thrombocytopenia
- •inability to access intrathecal space by CT or flouro guided injection
研究组 & 干预措施
Subjects with spinal muscular atrophy types 2 and 3
Intrathecal nusinersen will be administered to all subjects per FDA approved label.
干预措施: nusinersen (Drug)
结局指标
主要结局
Change in upper limb motor function
时间窗: Two years
Change from baseline to end of study in upper limb module score
Change in 6 minute walk test in ambulatory patients
时间窗: Two years
Change in 6 minute walk test from baseline to end of study
Change in quantitative motor function
时间窗: Two years
Primary Endpoint: Change from baseline to end of study in quantitative motor strength by dynamometry of upper limb muscles - shoulder abduction and elbow flexion/extension.
次要结局
- Change in compound muscle action potential (CMAP) amplitude by nerve conduction velocity (NCV) criteria(Two years)
- Change in pulmonary function by spirometry forced expiratory volume (FEV)(Two years)
- Change in pulmonary function by spirometry forced vital capacity (FVC)(Two years)
- Change in 10 meter walk test in ambulatory patients(Two years)