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: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: University of Florida
• Collaborators: Food and Drug Administration (FDA); Medosome Biotec LLC

Criteria

Inclusion Criteria:

• Healthy as outline in the physical exam and blood tests

• Non smoker

Exclusion Criteria:

• Cannot comprehend or refuse to sign the informed consent form;

• Febrile or have other clinical signs of infection;

• Pregnant or are nursing;

• In females, cannot or refuse to use contraception or avoid unprotected intercourse during the study;

• Uncontrolled hypertension (BPs > 160 mmHg or BPd > 100 mmHg) on conventional medication;

• Anemic (hematocrit < 35% in males; < 35% in females;

• Serum creatinine ≥ 1.3 mg/dl, TSH > 4.5 mIU/ml; a transaminase (ALT or AST) > 2 x ULN, total bilirubin > 1.2 mg/dl or fasting glucose ≥ 110 mg/dl.

• History of psychosis, seizures or diabetes mellitus or be receiving anti-psychotic, anti-epileptic or blood glucose-lowering medication.