NCT00004493

Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: University of California, San Diego · Academic / Other
• Sex: All
• Age: 3 Months
• Healthy volunteers: Not accepted

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

Conditions

• Lactic Acidosis

Interventions

Type	Name	Description
DRUG	sodium dichloroacetate

Timeline

• Start date: 1998-09-01
• Completion: 2003-09-01
• First posted: 1999-10-19
• Last updated: 2015-03-25

Locations

1 site across 1 country: United States