Safety and Tolerability of Coenzyme Q10 in Adult-Onset Sporadic Spinocerebellar Ataxia

This is a Physician-sponsored pilot study, whose purpose it is determine if high-dose oral Coenzyme Q10 (CoQ10) is safe and tolerated in patients with sporadic forms of adult-onset spinocerebellar ataxias (SAOA), a group of degenerative neurological disorders affecting the cerebellum and pathways to and from the cerebellum, with or without additional central nervous system (CNS) manifestations, in the absence of family history of degenerative ataxias.

CoQ10 is an essential cofactor of the electron transport chain and is a potent free radical scavenger in lipid and mitochondrial membranes. CoQ10 has shown efficacy in treatment of Parkinson's disease patients, and a Huntington's disease trial gave a trend of slowing down disease progression (CARE-HD). A small trial of CoQ10 in patients with Friedreich's ataxia suggested potential beneficial effects on ventricular thickness. CoQ10 is being tested on ALS patients and is considered to be potentially useful for treatment of Alzheimer's disease. Thus, CoQ10 is considered to be a promising therapeutic agent that might slow down the disease progression in a wide variety of neurodegenerative disorders. To date, very high doses of CoQ10 have not been used in patients with ataxia and the safety and tolerability in this group of patients should be established before efficacy trials are launched.

Twenty patients with SAOA will be recruited for a double-blind, randomized, placebo-controlled, multicenter study. Fifteen patients will receive a total of 2400 mg of oral CoQ10 daily, and five patients will receive placebo, for a period or 4 weeks. Cerebellar functions will be measured using a validated rating scale (SARA), an oculomotor examination, and functional measurement of motor function using a 9 hole peg test and timed walk. Safety labs will be collected and a digital movie will be recorded at the beginning (prior to treatment with CoQ10) and at the end of the study.