CLINICAL TRIAL OF HIGH DOSE COQ10 IN ALS

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。肌萎缩性侧索硬化症（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，而且无法治愈。氧化应激和线粒体功能障碍与ALS的病理生理有关。辅酶Q10 （CoQ10）是一种线粒体辅助因子和强大的抗氧化剂，可以延长转基因ALS小鼠模型的存活时间，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。因此，我们建议开展一项2期、随机、安慰剂对照、双盲、多中心的高剂量辅酶q10与安慰剂对照的II期临床试验（目标纳入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).