Metformin for the Prevention of Oral Cancer in Patients With Oral Leukoplakia or Erythroplakia

Running, not enrolling · Phase 2 · Has a placebo group

Conditions studied: Erythroplakia, Oral Leukoplakia

In brief

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.

Key facts

Study ID: NCT05237960
Run by: University of Arizona
People needed: 34
Starts: 2023-01-12
Expected to finish: 2026-12-01
Last updated by the study team: 2026-04-20

Who can join

Age: 21 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Participants with oral leukoplakia or erythroplakia with mild, moderate, or severe histologic dysplasia or hyperplasia at the high risk sites (e.g., floor of mouth, tongue). Lesions arising from the radiation field are excluded as study lesions.
Measurable disease - minimum lesion size of 8x3 mm before initial biopsy
Age >= 21 years. Adults 18-20 are not included as Canadian law prohibits purchase of cigarettes under the age of 21; investigators wish to keep criteria consistent among all trial sites. Also, smokers aged < 20 years would most likely not have oral leukoplakia
Current and former smokers (>= 5 packs in the lifetime)
Karnofsky performance scale >= 70%
Leukocytes >= 3,000/microliter
Absolute neutrophil count >= 1,000/microliter
Platelets >= 100,000/microliter
Total bilirubin =< 1.5 x institutional upper limit of normal (ULN)
Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 1.5 x institutional ULN
Estimation glomerular filtration rate (eGFR) > 45 mL/min (eGFR calculated using the equation Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] creatinine)
Willing to use adequate contraception (barrier method, abstinence, subject or partner has had a vasectomy or partner is using effective birth control or is postmenopausal) for the duration of study participation because the effects of metformin on the developing human fetus are unknown even though it is not teratogenic in rats and rabbits at 2-6 times the maximum recommended human daily dose. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her study physician immediately.
Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
Patients on chronic suppressive antiviral therapy for herpes simplex virus (HSV) are eligible
Ability to take oral medication
Ability to understand and the willingness to sign a written informed consent document in English or Spanish

You may not qualify if…

Patients with diabetes who are being treated with insulin or an anti-diabetic medication
History of diabetic ketoacidosis
Participants may not be receiving any other investigational agents within past 3 months at screening
History of allergic reactions attributed to compounds of similar chemical composition to metformin or prior use of metformin within the last year
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, human immunodeficiency virus (HIV) positive, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
Oral carcinoma in situ from the baseline biopsy
History of chronic alcohol use or abuse defined as any one of the following: a) average consumption of 3 or more alcohol containing beverages daily in the past 12 months; b) consumption of 7 or more alcoholic beverages within a 24 hour (hr) period in the past 12 months
Hemoglobin A1c (HbA1c) > 8%
Pregnancy or nursing women. Pregnant women are excluded from this study because the effects of metformin on the developing human fetus are unknown even though it is not teratogenic in rats and rabbits at 2-6 times the maximum recommended human daily dose. Because there is an unknown but potential risk for AEs in nursing infants secondary to treatment of the mother with metformin, breastfeeding should be discontinued
Acute or chronic liver disease, evidence of hepatitis (infectious or autoimmune), cirrhosis or portal hypertension
History of renal disease
Have received hormone therapy, chemotherapy, immunotherapy and/or radiation for any malignancy (excluding non-melanoma skin cancer and cancers confined to organs with removal as only treatment) within the past 18 months. History of prior curatively treated cancer, including oral cancer, is allowed as long as all primary and adjuvant treatment is completed >= 18 months prior to enrollment. Ongoing adjuvant hormonal treatment (e.g., for breast cancer) is allowed.
Current use of carbonic anhydrase inhibitors (e.g. topiramate, zonisamide, acetazolamide, or dichlorphenamide) or ranolazine

Where it is running

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

Full record on ClinicalTrials.gov

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