Pioglitazone-Metformin Combination Treatment for High Risk Oral Preneoplasia

Summary

This is a Phase IIa oral cavity leukoplakia study of pioglitazone 15mg and metformin 500mg BID for 12 weeks. The primary objective is to determine the clinical and histologic changes of leukoplakia from baseline following a 12 week course of twice daily pioglitazone-metformin. Outcomes are defined as are a reduction of the leukoplakia grade in \> 50% of treated participants and a partial or complete clinical response defined as 50% or greater reduction in the sum of measured targeted lesions. In addition, participants who show clinical and histologic improvement should correlate with a significant reduction of Ki-67 proliferative indices in lesions of these participants as compared to baseline.

Timeline

Start

2025-01-01

Primary completion

2026-07-28

Completion

2026-07-28

Publications

• Gates JC, Abouyared M, Shnayder Y, Farwell DG, Day A, Alawi F, Moore M, Holcomb AJ, Birkeland A, Epstein J. Clinical Management Update of Oral Leukoplakia: A Review From the American Head and Neck Society Cancer Prevention Service. Head Neck. 2025 Feb;47(2):733-741. doi: 10.1002/hed.28013. Epub 2024 Nov 25.
• Puri A, Wuertz B, Rhodus NL, Ondrey FG. Safety of oral mucosal punch biopsy and other oral biospecimen collections in clinical research. Oral Surg Oral Med Oral Pathol Oral Radiol. 2025 Mar;139(3):344-351. doi: 10.1016/j.oooo.2024.10.084. Epub 2024 Oct 30.

Drugs

Indications