Holter of Movement in Patients with Type 2 or 3 Spinal Muscular Atrophy When Initiating Treatment with Spinraza ° or Risdiplam. Acti-SMA Study.
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 5
- 主要终点
- 95th centile of stride velocity
研究概览
简要总结
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam. First, we want to quantify improvement of both ambulant and non-ambulant patients under treatment. A secondary objective would also be to identify suitable accelerometric measurements that are sensitive to change but also well correlated to other clinical scores.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed spinal muscular atrophy.
- •Treatment by Spinraza ° or by risdiplam planned.
- •Over 6 years old.
- •Signed informed consent.
排除标准
- •Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.
结局指标
主要结局
95th centile of stride velocity
时间窗: 39 months
95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life. (meter per second).
50th centile of stride velocity
时间窗: 39 months
50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).
95th centile of stride length
时间窗: 39 months
95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Upper limb function evolution in patient with SMA undergoing treatment
时间窗: 39 months
Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life.
50th centile of stride length
时间窗: 39 months
50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
次要结局
- 6-minute walk test(39 months)
- Dynamometric measures(39 months)
- 10-metre walk test(39 months)
- revised upper limb module (RULM)(39 months)
- 4-stair climb(39 months)
- Hamersmith funcitonnal motor scale (HFMS)(39 months)
- rise from floor(39 months)
- motor function measure (MFM)(39 months)
研究者
Margaux POLEUR
Doctor
Centre Hospitalier Universitaire de Liege