HIGH DOSE COQ10 IN ALS

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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.