Clinical Trial of High Dose CoQ10 in ALS

Amyotrophic lateral sclerosis (ALS) is a progressive and devastating neurodegenerative disorder. Available treatment for ALS remains scarce. Oxidative stress and mitochondrial dysfunction have been implicated in the pathophysiology of ALS. Oxidative stress refers to the effects of cell-damaging reactive oxygen species, also known as free radicals. Oxidative stress is thought to contribute to nerve cell loss in ALS. Mitochondria are organelles within each cell that are sometimes called "powerhouses of the cell" because cellular energy metabolism is located within the mitochondria.

Coenzyme Q10 (CoQ10), a mitochondrial cofactor known for its antioxidant properties, has prolonged survival in the mouse model of ALS and has slowed functional decline in another neurodegenerative disorder, Parkinson's disease. The goals of this double-blind, placebo-controlled, two-dose comparison phase II study are to obtain preliminary efficacy data and to select the preferred dose for a larger phase III study.

Participants were randomly assigned to CoQ10 (at two different dose levels) or placebo in the first stage, then the 2,700 mg dose was selected in the second stage. Duration of the trial was 9 months with a total of 7 visits.

Inclusion criteria

• Clinical diagnosis of definite, probable, or laboratory-supported probable ALS
• Negative pregnancy test for women of childbearing age and adequate birth control measures
• Subjects must be able and willing to give informed consent and must be capable of complying with the trial procedures
• Forced Vital Capacity (FVC) >/= 60% of predicted
• Age 21 to 85 years, inclusive
• Disease duration of less than 5 years
• Subjects may take riluzole (without change in dose for more than 30 days before enrollment)
• Patients who have taken CoQ10 in the past will be eligible if they stop at least 30 days before enrollment
• Patients who have taken vitamin E in the past will be eligible if they stop at least 14 days before enrollment

Exclusion criteria

• Dependency on mechanical ventilation (non-invasive ventilation > 23 hours)
• Severe and unstable concomitant medical or psychiatric illness
• Insufficiently controlled diabetes mellitus
• Concomitant warfarin therapy
• Women who are breast feeding or have a high likelihood of pregnancy
• Significant hepatic dysfunction
• Forced Vital Capacity (FVC) less than 60%
• Exposure to CoQ10 within 30 days of enrollment
• Exposure to other experimental medications within 30 days of enrollment
• Exposure to vitamin E within 14 days of enrollment
• Sensitivity to color additive FD&C Yellow No. 5
• Sensitivity to aspirin