Clinical Trial of High Dose CoQ10 in ALS
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 185
- 试验地点
- 19
- 主要终点
- Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
研究概览
简要总结
The purpose of this study is to determine the efficacy and preferred dose of CoQ10 in individuals with ALS for a possible future phase III study.
详细描述
Amyotrophic lateral sclerosis (ALS) is a progressive and devastating neurodegenerative disorder. Available treatment for ALS remains scarce. Oxidative stress and mitochondrial dysfunction have been implicated in the pathophysiology of ALS. Oxidative stress refers to the effects of cell-damaging reactive oxygen species, also known as free radicals. Oxidative stress is thought to contribute to nerve cell loss in ALS. Mitochondria are organelles within each cell that are sometimes called "powerhouses of the cell" because cellular energy metabolism is located within the mitochondria.
Coenzyme Q10 (CoQ10), a mitochondrial cofactor known for its antioxidant properties, has prolonged survival in the mouse model of ALS and has slowed functional decline in another neurodegenerative disorder, Parkinson's disease. The goals of this double-blind, placebo-controlled, two-dose comparison phase II study are to obtain preliminary efficacy data and to select the preferred dose for a larger phase III study.
Participants were randomly assigned to CoQ10 (at two different dose levels) or placebo in the first stage, then the 2,700 mg dose was selected in the second stage. Duration of the trial was 9 months with a total of 7 visits.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 21 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of definite, probable, or laboratory-supported probable ALS
- •Negative pregnancy test for women of childbearing age and adequate birth control measures
- •Subjects must be able and willing to give informed consent and must be capable of complying with the trial procedures
- •Forced Vital Capacity (FVC) >/= 60% of predicted
- •Age 21 to 85 years, inclusive
- •Disease duration of less than 5 years
- •Subjects may take riluzole (without change in dose for more than 30 days before enrollment)
- •Patients who have taken CoQ10 in the past will be eligible if they stop at least 30 days before enrollment
- •Patients who have taken vitamin E in the past will be eligible if they stop at least 14 days before enrollment
排除标准
- •Dependency on mechanical ventilation (non-invasive ventilation > 23 hours)
- •Severe and unstable concomitant medical or psychiatric illness
- •Insufficiently controlled diabetes mellitus
- •Concomitant warfarin therapy
- •Women who are breast feeding or have a high likelihood of pregnancy
- •Significant hepatic dysfunction
- •Forced Vital Capacity (FVC) less than 60%
- •Exposure to CoQ10 within 30 days of enrollment
- •Exposure to other experimental medications within 30 days of enrollment
- •Exposure to vitamin E within 14 days of enrollment
- •Sensitivity to color additive FD&C Yellow No. 5
- •Sensitivity to aspirin
研究组 & 干预措施
2,700 mg CoQ10
干预措施: coenzyme Q10 (Drug)
placebo
干预措施: Placebo (Drug)
1,800 mg CoQ10
干预措施: coenzyme Q10 (Drug)
结局指标
主要结局
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
时间窗: 9 months
The ALSFRSr, a questionnaire-based scale assessing daily living function ranging from 48 (best score) to 0 (worst), was administered to the patient, or to a proxy if the patient could not communicate effectively. Decline was defined as ALSFRSr at baseline minus ALSFRSr at month 9. Thus a positive value indicates worsening.
次要结局
- Change in Fatigue Severity Scale(9 months)
- Change in Forced Vital Capacity(9 months)
- Change in Short Form (SF)-36 Score (Physical)(9 months)
- Change in Short Form (SF)-36 Score (Mental)(9 months)