Clinical trial · Interventional
Metformin Hydrochloride in Preventing Oral Cancer in Patients With an Oral Premalignant Lesion
M4OC-Prevent: Metformin for Oral Cancer Prevention
NCT02581137CI-TRIAL-00116088active not recruitingPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This phase IIa trial studies how well metformin hydrochloride works in preventing oral cancer in patients with an oral premalignant lesion (oral leukoplakia or erythroplakia). Oral premalignant lesions look like red or whitish plaques or lesions in the mouth that do not rub off and can be associated with a higher risk of cancer. Metformin hydrochloride may help prevent oral cancer from forming in patients with an oral premalignant lesion.
Conditions
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Erythroplakia | — | UNRESOLVED | — |
| Hyperplasia | — | UNRESOLVED | — |
| Oral Cavity Carcinoma | Oral Cavity Carcinoma | ONTOLOGY_EXACT | 0.98 |
| Oral Leukoplakia | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Laboratory Biomarker Analysis | Other | — | UNRESOLVED |
| Metformin Hydrochloride | Drug | Metformin | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Prevention (extended-release metformin hydrochloride)
- description
- Patients receive extended-release metformin hydrochloride PO QD for 2 weeks and then BID for 10-12 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
- interventionNames
- Other: Laboratory Biomarker Analysis
- Drug: Metformin Hydrochloride
Primary outcomes (1)
- measure
- Clinical Response to Metformin Intervention
- timeFrame
- Baseline to up to 14 weeks
- description
- Number of participants with complete and partial clinical response to metformin intervention. Criteria for complete and partial clinical response are: Complete Response (CR): Disappearance of all evidence of lesion(s). Partial Response (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.
Secondary outcomes (7)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (1)
- DERIVEDGutkind JS, Molinolo AA, Wu X, Wang Z, Nachmanson D, Harismendy O, Alexandrov LB, Wuertz BR, Ondrey FG, Laronde D, Rock LD, Rosin M, Coffey C, Butler VD, Bengtson L, Hsu CH, Bauman JE, Hewitt SM, Cohen EE, Chow HS, Lippman SM, Szabo E. Inhibition of mTOR signaling and clinical activity of metformin in oral premalignant lesions. JCI Insight. 2021 Sep 8;6(17):e147096. doi: 10.1172/jci.insight.147096. PMID 34255745