CLINICAL TRIAL OF HIGH DOSE COQ10 IN ALS

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心机构，不一定为研究者机构。肌萎缩侧索硬化症（ALS）是最具破坏性的神经系统疾病之一，通常在发病3年内导致死亡。治疗非常有限，而且没有治愈方法。氧化应激和线粒体功能障碍与ALS的病理生理学有关。辅酶Q10（CoQ 10）是一种线粒体辅因子和强大的抗氧化剂，可延长转基因ALS小鼠模型的存活率，并减缓其他人类神经退行性疾病的功能下降。辅酶Q10是一种非处方膳食补充剂，具有良好的安全性和中枢神经系统渗透性。因此，我们建议进行一项2阶段、II期、随机、安慰剂对照、双盲、多中心的高剂量辅酶Q10与安慰剂的临床试验（目标招募18-20个临床站点的185名患者）。

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. Amyotrophic lateral sclerosis (ALS) is one of the most devastating neurological diseases leading to death typically in 3 years of onset. Treatment is severely limited, and no cure is available. Oxidative stress and mitochondrial dysfunction have been implicated in ALS pathophysiology. Coenzyme Q10 (CoQ10), a mitochondrial cofactor and powerful antioxidant, prolongs survival in the transgenic ALS mouse model and slows functional decline in other human neurodegenerative disease. CoQ10 is a nonprescription dietary supplement with an excellent safety profile and central nervous system penetration. Thus, we propose to conduct a 2-stage, phase II, randomized, placebo-controlled, double-blind, multicenter clinical trial of high dose CoQ10 against placebo (target enrollment 185 patients at 18-20 clinical sites).