Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia
Status:
Completed
Trial end date:
2003-09-01
Target enrollment:
Participant gender:
Summary
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.