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 ]
Current Secondary Outcome: DCA trough level [ Time Frame: Bi-annual evaluation ]
Original Secondary Outcome: Genotype [ Time Frame: up to 5 years ]
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