drugset / Trial / NCT02690285
A Pilot Study of Rapid Haplotyping Procedure for Personalized Dosing of Dichloroacetate (DCA) in Healthy Volunteers
Phase 1
Completed
45 enrolled
University of Florida
Food and Drug Administration (FDA) · collabMedosome Biotec LLC · collab
Non-randomizedSingle-groupOpen-labelScreening
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.
Timeline
- Start
- 2016-03-01
- Primary completion
- 2017-05-30
- Completion
- 2017-11-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Dichloroacetate | Small molecule | 25 mg/kg | Oral |
Indications
No indication recorded.