Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 50
- 主要终点
- Changes from baseline in pulmonary capacity of ALS patients at 6 months.
研究概览
简要总结
Amyotrophic lateral sclerosis (ALS) is a fatal disease leading to motor neuron degeneration and progressive paralysis. Other studies have revealed defects in skeletal muscle even in absence of motor neuron anomalies, focusing on acetylcholine receptors (AChRs) and supporting the so-called "dying-back" hypothesis. Outcome of this study will be to understand if the endocannabinoid palmitoylethanolamide (PEA) can reduce the rundown of AChRs currents in ALS muscle, and if it can modify ALS patients' clinical and electrophysiological parameters.
详细描述
Outcome:
Monitoring the efficacy and safety of PEA in the treatment of patients with ALS. Analysis of AChR currents and description of the composition of AChRs subunits in ALS muscles
Design of the Study:
A randomized controlled blinded study. Patients with sporadic ALS will receive riluzole alone or riluzole+PEA in order to investigate the clinical and electrophysiological effects of treatment. The expected number of enrolled patients will be 50.
All patients satisfying the selection criteria will be randomized into two groups: a first group will be treated only with riluzole, the second group with riluzole associated with PEA (Normast 600 mg microgranular, 2 sachets/day). The randomization will be done stratifying patietns according to type of clinical onset (bulbar vs. spinal). The patients will be enrolled in the Department of Neurology and Psychiatry, University of Rome "Sapienza".
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of ALS according to the El-Escorial criteria;
- •Age> 18 years;
- •ALS Functional Rating Scale-Revised (ALSFRS- r) score> 20;
- •Forced Vital Capacity (FVC)> 30%;
- •Treatment with Riluzole.
排除标准
- •Other diseases motor neurons;
- •Experimental treatments in the previous three months;
- •Pregnant or breast-feeding;
- •Contraindications to the use of riluzole;
- •Patients undergoing tracheostomy, enteral or parenteral supply;
- •Severe psychiatric disorders.
研究组 & 干预措施
Riluzole
Riluzole 50 mg twice daily in ALS patients
干预措施: Riluzole (Drug)
PEA plus Riluzole
Riluzole 50 mg twice daily plus Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily in ALS patients
干预措施: endocannabinoid palmitoylethanolamide (PEA) (Drug)
PEA plus Riluzole
Riluzole 50 mg twice daily plus Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily in ALS patients
干预措施: Riluzole (Drug)
结局指标
主要结局
Changes from baseline in pulmonary capacity of ALS patients at 6 months.
时间窗: six months
Changes of the percentage of predicted forced vital capacity (FVC %) will be measured
次要结局
- Changes in acetylcholine receptors (AChR) currents and Analysis of the composition of AChRs subunits in ALS muscles.(six months)
- Changes from baseline in muscle strength of ALS patients at 6 months.(six months)
- Changes from baseline in electrophysiological parameters of ALS patients at 6 months(six months)
研究者
Maurizio Inghilleri
Associated Professor of Neurology
University of Roma La Sapienza