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.

Condition	Treatment or Intervention	Phase
Lactic Acidosis	Drug: sodium dichloroacetate	Phase II

Further Study Details: 

Expected Total Enrollment:  75

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.

Ages Eligible for Study:  3 Months and above,  Genders Eligible for Study:  Both

Criteria

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

California
      University of California San Diego School of Medicine, La Jolla,  California,  92093-0652,  United States; Recruiting

Study chairs or principal investigators

Bruce Barshop,  Study Chair,  University of California, San Diego   

Study ID Numbers:  199/14274; UCSD-FDR001481
Record last reviewed:  May 2000
Last Updated:  October 13, 2004
Record first received:  October 18, 1999
ClinicalTrials.gov Identifier:  NCT00004493
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08