CLINICAL TRIAL OF HIGH DOSE COQ10 IN ALS

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。目的：该研究的主要目的是确定辅酶Q10是否能减轻ALS患者的功能衰退。这将分两个阶段进行。第一阶段将确定两种剂量(1800毫克或2700毫克)的辅酶Q10中哪一种更可取，第二阶段将所选剂量与安慰剂进行比较，以评估疗效。研究计划：这是一项分两个阶段的II期随机、安慰剂对照、双盲、多中心临床试验，高剂量、可溶解的辅酶Q10与安慰剂对照。方法：研究分为两个阶段。在第一阶段，总共105名患者将被随机分为三组：辅酶Q10 2700毫克、辅酶Q10 1800毫克或安慰剂。在基线和筛查访问后的九个月内，患者将每隔一个月接受一次检查。每次探视都要进行安全实验室、生命体征、呼吸和功能测试。在第一阶段之后，将进行为期三个月的收尾、数据和统计分析。第二阶段在分析选定的辅酶Q10剂量后开始。在第二阶段，80名患者将被分成两组：选定的辅酶Q10剂量和安慰剂。这一阶段将遵循第一阶段的模式，在九个月内每隔一个月访问一次患者，并使用相同的测试和评估模式。此外，来自第一阶段的70名患者的数据将用于分析第二阶段的结果。相关性：到目前为止，唯一登记的ALS药物治疗是利鲁唑，它将患者的生存期平均延长约三个月。任何有效地减轻ALS功能衰退或通过对抗运动神经元退化从而减缓ALS进展而改进现有治疗方法的治疗方法都将为ALS的理解和治疗提供重大进展。

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.