Overview

This trial is active, not recruiting.

Condition mitochondrial enzyme deficiencies
Treatment dichloroacetate
Phase phase 3
Sponsor University of Florida
Start date June 2005
End date June 2017
Trial size 3 participants
Trial identifier NCT01797276, IRB#201600336

Summary

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Other)
Patients are given the drug Dichloroacetate at a dose of 25mg/kg divided in two equal doses each day. They will take this drug for an indefinite period of time.
dichloroacetate DCA
Subjects are given DCA 25mg/kg in a divided dose twice a day.

Primary Outcomes

Measure
Nerve Conduction Study
time frame: Bi-annual evaluation
Liver function testing
time frame: Bi-annual evaluation

Secondary Outcomes

Measure
DCA trough level
time frame: Bi-annual evaluation

Eligibility Criteria

Female participants from 18 years up to 50 years old.

Inclusion Criteria: - Diagnosis of congenital lactic acidosis - Participation in Study: #183-1992: Dichloroacetate Treatment of Congenital Lactic Acidosis Exclusion Criteria: - Intolerance to DCA

Additional Information

Official title Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Open Label
Principal investigator Peter W. Stacpoole, PhD, MD
Description 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).
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by University of Florida.