Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Change from Baseline in the Motor Function Measure Scale
研究概览
简要总结
There is a general physiological rule that any organ or system needs some minimal amount of activity to prevent its atrophy or degeneration. Although the relevance of that rule to exercises in neuromuscular patients and for SMA in particular is not definitely proven, clinical observations seem to support this assumption. Also there are several experimental studies which provide additional support for utility of exercise for SMA.
However, making regular exercises may be very challenging with SMA not only due to physical limitations, but due to psychological either.
While being considered as safe and well tolerated intervention, TMS is able to mimic effects of real physical exercises, at least at the level of low motoneuron, it also provides several advantages. For example, possibility to exercise non-collaborative infants, minimization of psychological motivation impact in adults and/or ability to involve very weak muscle groups.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has a diagnosis of 5q-autosomal recessive SMA confirmed by DNA test.
- •Informed consent.
- •A minimum score of 1 for Entry Item "A" of the Revised Upper Limb Module (RULM) scale for SMA: "Can use hands to hold pencil or pick up a coin/token or drive a powered chair, use phone key pad"
排除标准
- •Subject has severe joint contractures that would affect ability to perform study measures, determined by the study physician.
- •Subject has a deconditioned respiratory system, per the discretion of the physician investigator.
- •Subject has behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator.
研究组 & 干预措施
TMS arm
Stimulation (HF-rTMS) of the primary motor cortex, M1 area of the limbs, at a frequency above 5 Hz and an intensity of 90-100% of the resting motor threshold (RMT), over 10 sessions, up to 2400 stimuli per session.
干预措施: High-frequency repetitive transcranial magnetic stimulation (Device)
TMS arm
Stimulation (HF-rTMS) of the primary motor cortex, M1 area of the limbs, at a frequency above 5 Hz and an intensity of 90-100% of the resting motor threshold (RMT), over 10 sessions, up to 2400 stimuli per session.
干预措施: lumbar puncture (Diagnostic Test)
结局指标
主要结局
Change from Baseline in the Motor Function Measure Scale
时间窗: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Motor Function Measure (MFM) scale measurement before and after TMS sessions
Change from Baseline of the Revised Upper Limb Module
时间窗: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Revised Upper Limb Module (RULM) scale measurement before and after TMS sessions
Change from Baseline of the Hammersmith Functional Motor Scale - Expanded
时间窗: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
Hammersmith Functional Motor Scale - Expanded (HFMSE) measurement before and after TMS sessions
Change from Baseline in the 6-Minute Walk Test
时间窗: The first assessment will be conducted before the initial TMS session, and the second will be performed one to two days after the final TMS session.
6-Minute Walk Test (6MWT) measurement before and after TMS sessions
次要结局
未报告次要终点