Could a common diabetes drug stop oral cancer before it starts?
NCT ID NCT02581137
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests whether metformin, a widely used diabetes drug, can prevent oral cancer in 26 people with precancerous mouth lesions (leukoplakia or erythroplakia). Participants take metformin by mouth, and researchers check if the lesions shrink or disappear. The goal is to see if this safe, affordable drug can stop cancer from forming.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Metformin (a common diabetes drug)
- What this could lead to
- If it works, this could point toward a way to prevent oral cancer in people with precancerous mouth lesions.
- What could go wrong
- This is a small, early-phase trial with only 26 participants, so results may not apply to everyone. Metformin is generally safe but can cause side effects like stomach upset.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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26 people
The number who actually took part.
- Started
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Jun 2016
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Participants with oral leukoplakia or erythroplakia with mild, moderate, or severe histologic dysplasia, or hyperplasia not associated with mechanical factors such as ill-fitted dentures * Measurable disease - minimum lesion size of 8 x 3 mm before initial biopsy * Karnofsky performance status \>= 70% * Leukocytes \>= 3,000/microliter * Absolute neutrophil count \>= 1,000/microliter * Platelets \>= 100,000/microliter * Total bilirubin =\< 1.5 × institutional upper limit of normal (ULN) * Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\<1.5 × institutional ULN * eGFR \> 40 mL/min using the Cockcroft-Gault equation * Life expectancy \> 3 months * Willing to use adequate contraception (barrier method, abstinence, subject has had a vasectomy or partner is using effective birth control or is postmenopausal) for the duration of study participation * Ability to take oral medication * Ability to understand and the willingness to sign a written informed consent document Exclusion Criteria: * Patients with diabetes who are taking insulin or oral agents * History of diabetic ketoacidosis * Participants may not be receiving any other investigational agents within past 3 months * 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 * 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 * Glycated hemoglobin (HbA1c) \> 8% * Pregnancy or nursing women * Acute or chronic liver disease, evidence of hepatitis (infectious or autoimmune), cirrhosis or portal hypertension * History of renal disease * History of prior head and neck squamous cell carcinoma (HNSCC) unless curatively treated for \>= 1 year * Have received chemotherapy and/or radiation for any malignancy (excluding non-melanoma skin cancer and cancers confined to organs with removal as only treatment) in the past 2 years; ongoing adjuvant hormonal therapy for breast cancer is allowed
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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BC Cancer Research Centre
Vancouver, British Columbia, V5Z 1L3, Canada
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UC San Diego Medical Center - Hillcrest
San Diego, California, 92103, United States
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University of British Columbia Hospital
Vancouver, British Columbia, V6T 2B5, Canada
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University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
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