Metformin for the Prevention of Oral Cancer in Patients With Oral Premalignant Lesions

NCT ID: NCT05536037

Last Updated: 2025-05-15

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-05-04
• Study Completion Date: 2024-03-16

Brief Summary

This phase I trial tests whether metformin works in reducing the annual transformation (development of invasive cancer) of oral precancerous lesions into cancerous lesions. Metformin is a drug approved for the treatment of diabetes, but studies have shown that it may have some anticancer properties. Giving metformin may help prevent or slow the development of oral cancer from precancerous lesions.

Detailed Description

PRIMARY OBJECTIVE:

I. To evaluate the transformation-free-survival in lesion types erythroplakia (EP) and verrucous hyperplasia (VH). 'Transformation' is defined as the development of invasive cancer.

SECONDARY OBJECTIVE:

I. To evaluate the current spontaneous regression rates, i.e., percentages of patients having lesion disappear within 1-year post treatment, in all four lesion types and compare them with historical documented regression rate in literature.

EXPLORATORY OBJECTIVE:

I. To evaluate the transformation-free-survival in lesion types homogenous leukoplakia (HL) and non-homogenous leukoplakia (NHL).

OUTLINE:

Patients receive metformin orally (PO) once daily (QD) on days 1-3 and then PO twice daily (BID) for up to 12 months in the absence of unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months for 24 months.

Conditions

Erythroplakia; Leukoplakia; Oral Cavity Carcinoma; Proliferative Verrucous Leukoplakia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Prevention (metformin)

Patients receive metformin PO QD on days 1-3 and then PO BID for up to 12 months in the absence of unacceptable toxicity.

Group Type: EXPERIMENTAL

Metformin

Intervention Type: DRUG

Given PO

Biopsy

Intervention Type: PROCEDURE

Undergo biopsy

Interventions

Metformin

Given PO

Intervention Type: DRUG

Biopsy

Undergo biopsy

Intervention Type: PROCEDURE

Other Intervention Names

1-Dimethylbiguanide; 657-24-9; N-dimethylbiguanide; N-Dimethylimidodicarbonimidic Diamide; BIOPSY_TYPE; Bx

Eligibility Criteria

Inclusion Criteria

• Age 18-85
• Clinical appearance of lesion

• Homogenous leukoplakia
• Non- homogenous leukoplakia
• Erythroplakia
• Proliferative verrucous leukoplakia
• Histologic appearance

• No dysplasia
• Mild dysplasia
• Moderate dysplasia
• Severe dysplasia
• Diabetics: if on metformin, will get them to 2000 mg per day, if not, will add metformin in consultation with endocrinologist
• All subjects must be able to comprehend and sign a written informed consent document
• Willing and able to be available for the duration of the study
• In general good health with no contraindication to biopsy or metformin therapy
• Laboratory results

Exclusion Criteria

• Carcinoma in-situ, verrucous carcinoma, invasive squamous cell carcinoma (SCCa)
• Exclude systemic causes of the lesion: pemphigus, pemphigoid, systemic lupus erythematosus (SLE), lichenoid drug reaction, human immunodeficiency virus (HIV), syphilis
• Exclude local inciting factors: rule out (r/o) but allowing 2 weeks to pass and see if there is resolution, if not and doesn't resolve with local measures, medical treatment, enroll

• Frictional: sharp tooth
• Trauma
• Immunosuppression by natural illness or medically induced
• Hypersensitivity or allergic reaction to metformin or some other contraindication

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Thomas Jefferson University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, United States

Countries

United States

Other Identifiers

JT 17064

Identifier Type: OTHER

Identifier Source: secondary_id

22D.296

Identifier Type: -

Identifier Source: org_study_id