Clinical Trial: Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title:
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 Summary:
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
Sponsor: University of California, San Diego
Current Primary Outcome:
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: FDA Office of Orphan Products Development
Dates:
Date Received: October 18, 1999
Date Started: September 1998
Date Completion: September 2003
Last Updated: March 24, 2015
Last Verified: May 2000