Reinnervation and Neuromuscular Transmission in Patients With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Blood biomarkers
研究概览
简要总结
The aim of this study is to describe the changes in the neuromuscular connection in patients with amyotrophic lateral sclerosis (ALS). The study consist of three substudies that have the following main hypothesis:
- that ALS patients do not demonstrate equal capacity for muscle reinnervation and that reinnervation preserves muscle function and thereby slows down progression.
- that blood concentrations of c-terminal agrin fragment (bCAF) reflect neuromuscular transmission deficiency and that blood concentration of neural cell adhesion molecule reflects degree of muscle denervation in patients.
- that ALS patients with decrement when examined with repetitive nerve stimulation have more physical fatigue, slower progression, higher degree of reinnervation and higher bCAF compared to ALS patients without decrement.
There will be 3 inclusion groups.
- patients referred for neurophysiological examination on suspicion of motor neuron disease.
- healthy controls
- disease control: patients with another motor neuron disease with slow progression.
All participants will be invited for at least 1 visit (baseline). If participants in group 1 eventually receive the diagnosis of ALS they will be invited for 2 additional visits 4 og 8 months after baseline visit, respectively.
Examinations will consist of:
- nerve conduction study
- repetitive nerve stimulation (except for healthy controls) to examine impairment of the neuromuscular connection.
- motor unit number estimation with MScanFit to estimate number and size of motor units.
- ultrasound examination of muscles to measure size and condition of muscles.
- questionnaires on fatigue and functional status.
- blood sample for measurement of specialized analysis (c-terminal agrin fragment and neural cell adhesion molecule) and routine analysis (liver and kidney function as well as neurofilament light chain)
- muscle strength assessment manually and by dynamometer to follow progression of muscle weakness
- bioelectrical impedance measurement to follow the overall body composition.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Referred to clinical neurophysiological examination on suspicion of motor neuron disease or diagnosed with ALS according to Gold Coast criteria within the last 3 months.
- •Age ≥18 years old
- •Able and willing to provide informed consent
排除标准
- •Former central or peripheral nervous system disease
- •Electrophysiological signs of polyneuropathy at baseline visit
- •Pacemaker
- •Pregnancy
- •For disease controls the exclusion criteria are the same, but the inclusion criteria:
- •Diagnosed with disease with slow, progressive loss of motor neurons
- •Age ≥18 years old
- •Able and willing to provide informed consent
结局指标
主要结局
Blood biomarkers
时间窗: Baseline
Difference in blood concentration of c-terminal agrin fragment and neural cell adhesion molecule at baseline between ALS patients, healthy controls and ALS mimic disease patients.
Reinnervation
时间窗: From baseline and 8 months
Difference between fast and slow progressing patients in change in mean amplitude size of motor units as estimated by MScanFit motor unit number estimation.
Fatigue and decrement
时间窗: Baseline
Difference in proportion of participants with decrement between ALS patients and ALS mimic disease patients as well as degree of fatigue among ALS patients with and without neuromuscular transmission deficiency.
次要结局
未报告次要终点