Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia
Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia
OBJECTIVES: I. Determine the pharmacokinetics of sodium dichloroacetate (DCA) in patients
 with congenital lactic acidemia.

 II. Determine the efficacy of DCA in decreasing the frequency and/or severity of acute
 episodes of acidotic illness, improving linear growth, improving neurological or
 developmental function, or slowing neurological or developmental deterioration in these
 patients.
PROTOCOL OUTLINE: This is a randomized, double blind, cross over study. Patients are
 randomized to start with one of two different doses of sodium dichloroacetate (DCA).

 Patients receive one of two doses of oral DCA for 6 months, then switch to the alternate dose
 for 6 months. This course is repeated once.

 Patients are followed for up to 2 years.

 Completion date provided represents the completion date of the grant per OOPD records
PROTOCOL ENTRY CRITERIA:

 --Disease Characteristics--

 -  Diagnosis of stable, persistent lactic acidemia Venous lactate at least 3 mM under
 basal conditions defined as: At least 4 hours postprandial No concurrent illness

 OR

 -  Diagnosis of cerebral lactic acidemia with elevated lactic acid in CSF but not in the
 blood

 -  No organic acidemias or defective gluconeogenesis

 --Patient Characteristics--

 -  Hematopoietic: Hemoglobin at least 7 mg/dL

 -  Hepatic: Bilirubin no greater than 3 times upper limit of normal (ULN) AST, ALT, or
 GGT no greater than 10 times ULN

 -  Renal: Creatinine no greater than 2 mg/dL

 -  Cardiovascular: Ejection fraction at least 25%

 -  Other: No hypoglycemia (blood sugar less than 50 mg/dL at no greater than 12 hours
 fasting) No severe peripheral neuropathy interfering with normal activities of living