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.
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||September 1998|
|Estimated Study Completion Date:||September 2003|
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004493
|United States, California|
|University of California San Diego School of Medicine|
|La Jolla, California, United States, 92093-0652|
|Study Chair:||Bruce Barshop||University of California, San Diego|