NCT05120284

Trial of Dichloroacetate (DCA) in Glioblastoma Multiforme (GBM)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: University of Florida · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

Conduct a multicenter, open label Phase IIA trial of oral DCA in 40 surgical patients with recurrent GBM who have clinically indicated debulking surgery planned. No patients will be recruited at UF. Patients will be genotyped to establish safe dosing regimens and will be randomized to receive DCA (N=20) or no DCA (N=20) for one week prior to surgery. Deidentified blood and tumor tissue obtained at surgery will be assessed at UF for biochemical markers of DCA dynamics.

Detailed description

Evaluate effects of dichloroacetate (DCA) on tumor PDC phosphorylation.

Conditions

• Glioblastoma Multiforme

Interventions

Type	Name	Description
DRUG	Dichloroacetate (DCA)	Study medication DCA is a liquid formulation mixed with an artificial sweetener containing aspartame and strawberry extract (50mg/mL) Participants will be genotyped to determine GSTZ1 (glutathione S-transferase Zeta-1) haplotype status, which will stratify this group into 1 of 2 dose regimens: EGT carriers will receive 12-14 mg/kg/12hr DCA. EGT non-carriers will receive 6-7 mg/kg/12 hr.
GENETIC	Genotype	Participants will be genotyped to determine GSTZ1 haplotype status.

Timeline

• Start date: 2022-07-01
• Primary completion: 2026-07-01
• Completion: 2026-08-31
• First posted: 2021-11-15
• Last updated: 2025-08-29

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study