OxiDative Stress Clinical Trial


Howard M. Lederman, MD, PhD


A-T Clinical Center, Johns Hopkins Hospital


This placebo controlled, cross-over trial was performed to determine the safety of, and identify laboratory markers for treatment with a combination of nicotinamide and the antioxidant alpha-lipoic acid, compounds that may slow the brain cell death associated with A-T.


200 mg of alpha-lipoic acid was administered three times per day and 12.5 mg/kg of nicotinamide was administered two times per day. For the first two months, participants were randomly selected to receive either a) alpha-lipoic acid + placebo for nicotinamide or b) nicotinamide + placebo for alpha lipoic acid, then each group “crossed over” or switched compounds for the next two months. For the final two months of the trial, all participants were treated with both compounds.

Two laboratory markers of oxidative stress significantly improved when participants took both alpha-lipoic acid and nicotinamide. A trend toward increased lymphocyte counts was also observed, but did not reach statistical significance. None of these changes was thought to be clinically important. However, these findings will form the basis for examination of other laboratory markers of oxidative stress in future trials of novel antioxidant treatments. In addition, this study allowed for the development of accurate and reproducible methods for measuring the lung function of patients with A-T which can also be used in future clinical studies or trials.

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