CLINICAL TRIAL: CLINICAL TRIAL OF HIGH DOSE COQ10 IN ALS

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。列出的机构是 中心，不一定是研究者的机构。 目的：主要研究目的是确定辅酶Q10是否可以减少ALS患者的功能下降。 这将分两个阶段进行。 第一阶段将确定两种剂量（1800 mg或2700 mg）中哪一种是优选的，第二阶段将所选剂量与安慰剂进行比较，以评估疗效。 研究报告：这是一个两阶段的II期，随机，安慰剂对照，双盲，多中心的高剂量，可溶性辅酶Q10对安慰剂的临床试验。 方法：本研究分为两个阶段。 在第一阶段，共有105名患者将被随机分配到三组之一：CoQ 10 2700 mg，CoQ 10 1800 mg或安慰剂。 在基线和筛选访视后，患者将每隔一个月接受一次访视，持续9个月。 每次访视时将进行安全性实验室检查、生命体征、呼吸和功能检查。 第1阶段之后，将进行为期3个月的收尾、数据和统计分析。 第2阶段开始后，分析选定的辅酶Q10剂量。 在第二阶段，80名患者将被分为两组：选择的辅酶Q10剂量和安慰剂。 该阶段将遵循第1阶段的格式，每隔一个月进行一次患者访视，持续9个月，并使用相同的测试和评价格式。 此外，第1阶段70例患者的数据将用于分析第2阶段结果。 相关性：迄今为止，唯一注册的ALS药物治疗是利鲁唑，其平均延长患者生存期约三个月。 任何有效减少ALS功能下降或通过抵消运动神经元变性从而减缓ALS进展而改善当前疗法的治疗将在理解和治疗ALS方面提供显著进步。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. OBJECTIVE: The primary study objective is to identify whether CoQ10 reduces functional decline in patients with ALS. This will be done in two stages. Stage 1 will identify which of two (1800 mg or 2700 mg) doses of CoQ10 is preferable and Stage 2 compares the selected dose against placebo to assess efficacy. RESEARCH PLAN: This is a two-stage phase II, randomized, placebo-controlled, double-blind, multicenter clinical trial of high dose, solubilized CoQ10 against placebo. METHODS: The study consists of two stages. In Stage 1, a total of 105 patients will be randomized to one of three groups: CoQ10 2700 mg, CoQ10 1800 mg or placebo. Patients will be seen every other month for nine months following baseline and screening visits. Safety labs, vital signs, breathing and functional tests will be done at each visit. Following Stage 1, there will be a close out, data and statistical analysis of three months. Stage 2 begins following analysis of the selected CoQ10 dose. During Stage 2, 80 patients will be placed in one of two groups: Selected CoQ10 dose and placebo. This stage will follow the format of Stage 1 with patient visits every other month for nine months and utilizing the same test and evaluation format. In addition, the data from 70 patients in Stage 1 will be utilized in analyzing Stage 2 results. RELEVANCE: To date the only registered pharmacological treatment for ALS is riluzole, which extends patients survival on average by approximately three months. Any treatment which is effective in reducing functional decline in ALS or improves on current therapies by counteracting the degeneration of motor neurons and consequently slowing the progression of ALS would provide a significant advance in the understanding and treatment of ALS.