Coenzyme Q10 in Adult-Onset Ataxia
Condition: Sporadic Ataxia
MeSH terms: Ataxia; Cerebellar Ataxia
Study type: Interventional
Phase: Phase 1

Summary: 
      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.
    

Detailed description: 
      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.
    

Intervention name(s): Placebo (sugar pill); Coenzyme Q10
Intervention type(s): Drug; Drug
Intervention description(s): placebo: 2 wafers 4 times a day; 300mg wafers; 2 wafers 4 times a day
Other intervention name(s): CoQ10
Intervention MeSH terms: Ubiquinone; Coenzyme Q10
Allocation: Randomized
Intervention model: Single Group Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Supportive Care
Keywords: adult onset ataxia