Sporadic Ataxia Clinical Trial
Official title:
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.
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.
;