Randomized, Parallel Safety and Efficacy Study of Lipoic Acid in Patients With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 150
- 试验地点
- 1
- 主要终点
- change of Amyotrophic lateral sclerosis Function Rate Scale-Revised (ALSFRS-R)
研究概览
简要总结
In this proposed study, the investigators will evaluate the safety and efficacy of lipoic acid in treatment of Amyotrophic lateral sclerosis (ALS). The study will recruit 150 AD patients, and then these patients will be randomized to lipoic acid group or control group (75 patients per arm) for 6 courses for about 5 months. Clinical assessment will be done at screen/baseline, 3th course and 6th course. The specific aims are to compare lipoic acid versus control on: motor function and disease progression. During the study period, clinical effect index will be recorded, including bulbar function, motor function, respiratory function, and safety index including blood and urine routine, liver and kidney function, coagulation function.
详细描述
In this proposed study, the investigators will evaluate the safety and efficacy of Lipoic acid in treatment of ALS. The study will recruit 150 ALS patients, then these patients will be randomized to lipoic acid group or control group (75 patients per arm) for 6 courses for about 5 months. Clinical efficacy and safety assessment will be done at screen/baseline, 3th course and 6th course. The specific aims are to compare lipoic acid versus placebo on: (1) Lipoic acid could improve the motor function, delay the disease progression and extend survival time in patients with ALS, measured by the ALSFRS-R Scale, ROADS Scale, upper motor neuron Scale, Muscle strength Scale and Electromyography; (2) Lung function will be collected to prove the hypothesis lipoic acid may help respiratory function. (3) Safety index including blood and urine routine, liver and kidney function, coagulation index will be recorded.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
outcomes assessors who do all the scales are concealed of group allocation.
入排标准
- 年龄范围
- 20 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age range from 20 to 75 (including 20 and 75 years old), regardless of ethnic group or gender;
- •The subjects should meet the diagnostic criteria for ALS by El Escorial revised criteria: "Definite ALS", "Probable ALS" and "Probable, laboratory-supported ALS".
- •ALS Functional Rating Scale-Revised (ALSFRS-R 12 items) each item score ≥2 points;
- •The onset (the symptoms of limbs weakness, muscle atrophy or bulbar involvement ) of the disease is less than 2 years
- •Baseline breath function: Forced Vital Capacity≥70% .
- •Disease Progression Rate FS=(48- ALSFRS-R at "time of diagnosis")/duration from onset to diagnosis (month), progression rate FS≤1;
排除标准
- •Combined with one of cerebrovascular disease, spinal cord disease, spinal muscular atrophy, juvenile myoatrophy of distal upper extremity, multifocal motor neuropathy, Kennedy disease, epilepsy, etc;
- •Severe renal insufficiency: creatinine clearance rate <30 mL/min (Cockcroft-Gault formula, urea nitrogen and (or) blood creatinine> 1.5 times the upper limit of normal, or other known severe renal insufficiency diseases;
- •Severe liver damage: ALT, AST> 3 times the upper limit of normal, or other known liver diseases such as acute and chronic hepatitis, cirrhosis, etc.;
- •Obvious tachycardia or bradycardia; patients with acute myocardial infarction or interventional therapy in the past 6 months (patients with grade III-IV according to NYHA classification);
- •Combined with malignant tumor, blood, digestion or other serious diseases;
- •Female patients during pregnancy and lactation;
- •Participated in other clinical trials within 30 days before randomization, or are participating in other clinical trials;
研究组 & 干预措施
lipoic acid group
The patients will take lipoic acid by intravenous. At the same time, the patients will take take riluzole tablets orally everyday.
干预措施: lipoic acid group (Drug)
control group
The patients will take riluzole tablets orally everyday.
干预措施: control group (Drug)
结局指标
主要结局
change of Amyotrophic lateral sclerosis Function Rate Scale-Revised (ALSFRS-R)
时间窗: change from baseline to 11th week& 21th week
The Amyotrophic lateral sclerosis Function Rate Scale-Revised (ALSFRS-R) will be performed to test the motor function of patients at the enrollment, 3th course and 6th course . The score ranges from 0 to 48#and higher scores represent a better motor function.
次要结局
- change of Rasch Overall ALS Disability Scale (ROADS)(change from baseline to 11th week& 21th week)
- change of Upper motor neuron scale (UMNS)(change from baseline to 11th week& 21th week)
- change of Muscle strength scale(change from baseline to 11th week& 21th week)
- change of Pulmonary Forced Vital Capacity (PFVC)(change from baseline to 11th week& 21th week)
- change of Motor Neuron Disease Electromyography(change from baseline EMG to 21th week)