Clinical Trial: Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Open Label

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Open Label

Brief Summary: Open Label access to DCA as continued treatment for congenital lactic acidosis.

Detailed Summary: Subjects who participated in a Phase 3 Randomized Controlled Trial of DCA for treatment of Congenital Lactic Acidosis (CLA) are eligible to continue treatment with investigational medication DCA at the same dose of 25mg/kg/day. Study participants must travel to the study site for bi-annual evaluation by the study investigator. Bi-annual evaluation will include an interim medical history review, physical exam, blood collection for DCA trough level, and urine pregnancy testing (if indicated).
Sponsor: University of Florida

Current Primary Outcome:

  • Nerve Conduction Study [ Time Frame: Bi-annual evaluation ]
    A standard of care nerve conduction study is completed every 6 months, the report is added to the study record and reviewed by the study investigator.
  • Liver function testing [ Time Frame: Bi-annual evaluation ]
    Reports from standard liver function blood tests are added to the study record and reviewed by the investigator.


Original Primary Outcome: Safety and Efficacy of Dichloroacetate in Patients with Congenital Lactic Acidosis [ Time Frame: up to 5 years ]

Determine the chronic safety of oral DCA in patients with CLA, as assessed by periodic clinical biochemical and nerve conduction evaluations.


Current Secondary Outcome: DCA trough level [ Time Frame: Bi-annual evaluation ]

Blood sample for DCA trough level is collected prior to the morning dose.


Original Secondary Outcome: Genotype [ Time Frame: up to 5 years ]

A secondary objective is to determine the genotype of the subjects. It has been determined that genotype affects the metabolism of dichloroacetate. There are slow metabolizers and fast metabolizers. It is important to know a person's genotype for dosing purposes. Patients will have genotypes done.


Information By: University of Florida

Dates:
Date Received: February 15, 2013
Date Started: June 2005
Date Completion: June 2017
Last Updated: May 24, 2016
Last Verified: May 2016