Metformin for the Prevention of Oral Cancer in Patients With Oral Premalignant Lesions
Metformin Chemoprevention of Oral Premalignant Lesions
Study Overview
Status
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
Study Locations
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, United States, 19107
- Thomas Jefferson University Hospital
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Prevention
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: 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.
|
Undergo biopsy
Other Names:
Given PO
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Transformation-free-survival, in lesion types erythroplakia and verrucous hyperplasia
Time Frame: Up to 3 years
|
Will be summarized using Kaplan-Meier curves as well as 95% confidence ands.
Kaplan-Meier estimates of the 3-year transformation-free-survival rates will be reported with 95% confidence interval.
One-sample two-sided log-rank test will be applied to all four lesions as well.
|
Up to 3 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Change in lesion status
Time Frame: Baseline to 1 year after treatment
|
Evaluated on a Likert-scale with three levels: 'worsen', 'same' and 'disappear', compared to each patient's baseline lesion status (as measured at pre-treatment and one year post initiation of treatment).
The lesion size and status evaluated at 1-year post-treatment will be summarized using percentages as well as 95% Clopper-Pearson exact confidence intervals.
The current spontaneous regression rates (i.e., percentage of 'disappear') will be compared against the historically documented regression rate of 35% using two-sided exact binomial test.
|
Baseline to 1 year after treatment
|
Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Transformation-free-survival, in lesion types homogenous leukoplakia and non-homogenous leukoplakia
Time Frame: Up to 3 years
|
Will be summarized using Kaplan-Meier curves as well as 95% confidence ands.
Kaplan-Meier estimates of the 3-year transformation-free-survival rates will be reported with 95% confidence interval.
One-sample two-sided log-rank test will be applied to all four lesions as well.
|
Up to 3 years
|
Collaborators and Investigators
Sponsor
Collaborators
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 22D.296
- JT 17064 (Other Identifier: JeffTrial Number)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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