Mitochondrial Enzyme Deficiencies Clinical Trial
Official title:
Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
Expanded access to DCA as continued treatment for congenital lactic acidosis.
Subjects who participated in a Phase 3 Randomized Controlled Trial of DCA for treatment of Congenital Lactic Acidosis (CLA) are eligible to continue treatment with investigational medication DCA at the same dose of 25mg/kg/day for expanded access. Study participants must travel to the study site for bi-annual evaluation by the study investigator. Bi-annual evaluation will include an interim medical history review, physical exam, blood collection for DCA trough level, and urine pregnancy testing (if indicated). ;