A Pilot Study of Rapid Haplotyping Procedure for Personalized Dosing of Dichloroacetate (DCA) in Healthy Volunteers
Status:
Completed
Trial end date:
2017-11-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to identify and analyze the frequency of GSTZ1 haplotypes in a
healthy adult population and determine the pharmacokinetics of Dichloroacetate (DCA)
metabolism based on haplotype analysis.
The DCA drug is the first targeted treatment for Pyruvate Dehydrogenase Complex Deficiency
(PDCD).
This pilot study, focuses on developing a high throughput, sensitive and accurate screening
test for determining glutathione transferase zeta 1 (GSTZ1) haplotype status in individuals
who would be treated with DCA.
Phase:
Phase 1
Details
Lead Sponsor:
University of Florida
Collaborators:
Food and Drug Administration (FDA) Medosome Biotec LLC