Longitudinal Assessment of Autonomic and Sensory Nervous System in ALS
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 4
- 主要终点
- Autonomic peripheral innervation
研究概览
简要总结
The goal of this interventional non-pharmacological study is to evaluate, using a multimodal approach, the progression of autonomic and sensory involvement in in amyotrophic lateral sclerosis (ALS) patients enrolled within 18 months from motor onset and its relationship with the progression of overall clinical disability.
The main questions it aims to answer are:
- Is autonomic dysfunction at diagnosis associated with disease progression and survival in patients with Amyotrophic Lateral Sclerosis ?
- Can we identify in the skin biomarkers to be used as reliable measures of disease progression and to apply in future clinical trials for patient stratification and to assess response to drug treatment ? Participants at time 0 will receive a full clinical and instrumental examination and a blood sample testing to check inclusion and exclusion criteria, genetic screening for the most common genes associated with ALS (SOD1, FUS, TARDBP and c9orf72), questionnaires about clinical characteristics, quality of life, pain and a multidomain battery of neuropsychological tests, multimodal assessment of the autonomic nervous system including skin biopsy for morphological study. At follow-up we'll perform clinical scales and skin biopsy.
Researchers will compare results from ALS patients with data obtained from a population of age and sex matched healthy subjects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- None
盲法说明
There are not masking participants
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
- •ALS patients will be recruited within 18 months from the motor symptoms onset
排除标准
- •glucose intolerance or conditions potentially affecting the peripheral nervous system
研究组 & 干预措施
Amyotrophic Lateral Sclerosis patients
Amyotrophic Lateral Sclerosis (ALS) patients within 18 months from symptoms onset will be recruited
干预措施: Skin biopsy (Diagnostic Test)
Amyotrophic Lateral Sclerosis patients
Amyotrophic Lateral Sclerosis (ALS) patients within 18 months from symptoms onset will be recruited
干预措施: Cardiovascular Reflexes testing (Diagnostic Test)
Amyotrophic Lateral Sclerosis patients
Amyotrophic Lateral Sclerosis (ALS) patients within 18 months from symptoms onset will be recruited
干预措施: Administration of clinical scales evaluating autonomic symptoms, pain small fiber neuropathy symptoms (Diagnostic Test)
Amyotrophic Lateral Sclerosis patients
Amyotrophic Lateral Sclerosis (ALS) patients within 18 months from symptoms onset will be recruited
干预措施: Dinamic Sweat Test (Diagnostic Test)
Healthy controls
A population of healthy controls matched for sex and age will be enrolled
干预措施: Skin biopsy (Diagnostic Test)
Healthy controls
A population of healthy controls matched for sex and age will be enrolled
干预措施: Cardiovascular Reflexes testing (Diagnostic Test)
Healthy controls
A population of healthy controls matched for sex and age will be enrolled
干预措施: Administration of clinical scales evaluating autonomic symptoms, pain small fiber neuropathy symptoms (Diagnostic Test)
Healthy controls
A population of healthy controls matched for sex and age will be enrolled
干预措施: Dinamic Sweat Test (Diagnostic Test)
结局指标
主要结局
Autonomic peripheral innervation
时间窗: At follow-up, an average of 6 months
Quantification sweat gland (fiber lenght/um3) and arrector pili muscle (ff/mm) innervation in skin biopsy from thigh.
Sensory peripheral innervation (IENF)
时间窗: At follow-up, an average of 6 months
Quantification Intraepidermal Nerve Fibers (IENF ff/mm) in skin biopsy from thigh.
Sensory peripheral innervation (IENF)
时间窗: At recruitment
Quantification Intraepidermal Nerve Fibers (IENF ff/mm) in skin biopsy from fingertip, thigh and leg.
Autonomic peripheral innervation
时间窗: At recruitment
Quantification of nerves in sweat gland (fiber lenght/um3) in skin biopsy from fingertip, thigh and leg. Quantification of nerves in arrector pili muscle (ff/mm) in skin biopsy from thigh and leg.
次要结局
- Assessment of Cardiovascular function(baseline)
- Sensory and autonomic symptoms evaluated by clinical scales(At follow-up, an average of 6 months)
- Sudomotor function(baseline)
- Sensory and autonomic symptoms evaluated by clinical scales(At the recruitment)