Controlled clinical trial of dichloroacetate for treatment of congenital lactic acidosis in children.

Objective: Open-label studies indicate that oral dichloroacetate (DCA) may be effective in treating patients with congenital lactic acidosis. We tested this hypothesis by conducting the first double-blind, randomized, control trial of DCA in this disease.

Methods: Forty-three patients who ranged in age from 0.