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。嗜肌性侧索硬化症(ALS)是一种破坏性的神经退行性疾病，治疗非常有限。氧化应激和线粒体功能障碍与其病理生理学有关。辅酶Q10(CoQ10)是一种线粒体辅因子，是一种强大的抗氧化剂，在转基因ALS小鼠模型中延长了存活时间，并减缓了除ALS以外的人类神经退行性疾病的功能衰退。我们提出了辅酶Q10与安慰剂对照的两阶段、II期、随机、安慰剂对照、双盲、多中心临床试验。第一阶段确定两种剂量的辅酶Q10中的哪一种是首选的。第二阶段将首选剂量与安慰剂进行比较，以评估是否有足够的有效性证据来证明进入第三阶段试验的合理性。

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. Amyorophic lateral sclerosis (ALS) is a devastating neurodegenerative disorder for which treatment is severly limited. Oxidative stress and mitochondrial dysfunction have been implicated in its pathophysiology. Coenzyme Q10 (CoQ10), a mitochondrial cofactor and powerful antioxidant, has prolonged survival in transgenic ALS mouse model and has slowed functional decline in human neurodegenerative disease other than ALS. We propose a two-stage, phase II, randomized, placebo-controlled, double-blind, multicenter clinical trial of coenzyme Q10 against placebo. The first stage identifies which of two doses of CoQ10 is preferred. The second stage compares the preferred dose against placebo to assess whether there is sufficient evidence of efficacy to justify proceeding to a phase III trial.