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Expanded access to DCA as continued treatment for congenital lactic acidosis.
Condition or disease
Mitochondrial Enzyme Deficiencies
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).
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Ages Eligible for Study:
18 Years to 50 Years (Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Diagnosis of congenital lactic acidosis
Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria: