Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Enrollment
- 75
- Locations
- 1
Study Overview
Brief 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.
Detailed Description
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
Study Design
- Study Type
- Interventional
- Primary Purpose
- Treatment
Eligibility Criteria
- Ages
- 3 Months to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion 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
- •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
Exclusion Criteria
- Not provided