This phase IIb trial tests whether metformin works in preventing oral cancer in patients with oral leukoplakia (white patches) or erythroplakia (red patches). Metformin is in a class of drugs called biguanides. Metformin helps to control the amount of glucose (sugar) in the blood. It decreases the amount of glucose patients absorb from food and the amount of glucose made by the liver. Metformin also increases the body's response to insulin, a natural substance that controls the amount of glucose in the blood. This trial may help researchers determine if metformin can stop changes in the mouth that are related to pre-cancer growths in the mouth.
PRIMARY OBJECTIVE: I. To determine the histological response to metformin hydrochloride (metformin) intervention in the target lesion. SECONDARY OBJECTIVES: I. Clinical response to metformin intervention in the target lesion. II. Effect of metformin on cell proliferation (Ki67) and its molecular targets (pS6 and nuclear YAP) in the target lesion. III. Metformin effect on serum metabolic markers (C-peptide, glucose and HbA1c). IV. Trough plasma metformin concentrations. EXPLORATORY OBJECTIVES: I. Expression of dysregulated molecular mechanisms in the target lesion, including, in order of priority, p53, PTEN, p16, EGFR, and pEGFR. II. Immune cell infiltration and markers of inflammation in the target lesion. III. Analysis of genomic alterations in the target lesion and blood deoxyribonucleic acid (DNA). IV. Microbiome in oral rinses. OUTLINE: Patients are randomized to 1 of 2 arms. ARM I: Patients receive extended release metformin hydrochloride orally (PO) once daily (QD) for 24 weeks in the absence of disease progression or unacceptable toxicity. Patients also undergo collection of blood and biopsy at baseline and week 24. ARM II: Patients receive a placebo PO QD for 24 weeks in the absence of disease progression or unacceptable toxicity. Patients also undergo collection of blood and biopsy at baseline and week 24. After completion of study treatment, patients are followed for up to 3 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
DOUBLE
Enrollment
34
University of Arizona Cancer Center-North Campus
Tucson, Arizona, United States
UC San Diego Medical Center - Hillcrest
San Diego, California, United States
Moffitt Cancer Center
Tampa, Florida, United States
Emory University Hospital/Winship Cancer Institute
Atlanta, Georgia, United States
Louisiana State University
Lafayette, Louisiana, United States
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, United States
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, United States
NYU College of Dentistry
New York, New York, United States
British Columbia Cancer Agency
Vancouver, British Columbia, Canada
Dalhousie University
Halifax, Nova Scotia, Canada
Histologic response to metformin
Histologic response will be evaluated by the following criteria: complete response (CR): Complete reversal of dysplasia or hyperplasia to normal epithelium in the target lesion. Partial response (PR): Improvement of the degree of dysplasia or hyperplasia in the target lesion. No change (NC): No change in the degree of dysplasia or hyperplasia in the target lesion, anything that is not CR, PR or PD. Progressive disease (PD): Increase in the severity of grade of histology in the target lesion.
Time frame: Up to 24 weeks
Clinical response to metformin
Clinical response will be evaluated by the following criteria: CR: disappearance of all evidence of lesion(s). PR: greater than or equal to 50% reduction in the sum of the products of diameters of lesion(s) measurable at baseline. Non-measurable lesion(s) may not increase greater than or equal to 25% in size and no new lesion may appear. NC: no change in the size of the lesion(s) identified at baseline and no new lesions appearing, i.e., anything that is not CR, PR, or PD. PD: any increase greater than or equal to 25% in the product of the diameters of any lesion(s) measurable at baseline or in the estimated size of lesion(s) nonmeasurable at baseline or the appearance of an unequivocal new lesion.
Time frame: Up to 24 weeks
Cell proliferation
Effect of metformin on cell proliferation (Ki67) and its molecular targets (pS6 and nuclear YAP) in the target lesion. The change (pre to post) will be compared between arms.
Time frame: Up to 24 weeks
Serum metabolic markers
Metformin effect on serum metabolic markers (C-peptide, glucose and HbA1c). The change (pre to post) in serum metabolic markers will be compared between arms.
Time frame: Up to 24 weeks
Plasma metformin concentrations
The plasma metformin concentrations will be determined in the pre- and post-intervention samples.
Time frame: From baseline, up to 24 weeks
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