ErythroplakiaPhase 1NCT05536037
Metformin for the Prevention of Oral Cancer in Patients With Oral Premalignant Lesions
A Phase 1 study testing Metformin and Biopsy in people with Erythroplakia and Leukoplakia, run by Thomas Jefferson University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Erythroplakia, Leukoplakia, Oral Cavity Carcinoma
- What is being tested?
- Metformin and Biopsy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 4 May 2022to 16 Mar 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18-85
- Clinical appearance of lesion
- Homogenous leukoplakia
- Non- homogenous leukoplakia
- Erythroplakia
- Proliferative verrucous leukoplakia
Show all 16Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
About this study
Official title: Metformin Chemoprevention of Oral Premalignant Lesions
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.
Read the detailed description
PRIMARY OBJECTIVE:
- 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:
- 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:
- 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.
- Being tested
- Metformin, Biopsy
- Medical subject headings
- Erythroplasia, Leukoplakia, Mouth Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetforminDrug
Also called 1-Dimethylbiguanide, 657-24-9, N-dimethylbiguanide, N-Dimethylimidodicarbonimidic Diamide
Given PO
BiopsyProcedure or surgery
Also called BIOPSY_TYPE, Bx
Undergo biopsy
| Study group | Role | What they receive |
|---|---|---|
| Prevention (metformin) | Experimental | Patients receive metformin PO QD on days 1-3 and then PO BID for up to 12 months in the absence of unacceptable toxicity. Receives: Drug: Metformin; Procedure: Biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Transformation-free-survival, in lesion types erythroplakia and verrucous hyperplasia
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.
Measured: Up to 3 years
Secondary outcomes (1)
Change in lesion status
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.
Measured: Baseline to 1 year after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2022
- Study start4 May 2022
- Primary completion16 Mar 2024
- Study completion16 Mar 2024
Why it stopped: Slow accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ErythroplakiaStudies of the same condition, open and under way.
- More trials run by Thomas Jefferson UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Thomas Jefferson University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05536037
- Sponsor's protocol ID
- 22D.296
- Other IDs
- JT 17064
- Status last verified
- May 2025
- First submitted
- 7 Sep 2022
- Last updated
- 15 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Metformin Chemoprevention of Oral Premalignant Lesions
- ClinicalTrials.gov ID
- NCT05536037
- Acronym / sponsor ID
- 22D.296
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow accrual
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Thomas Jefferson UniversityUniversity, hospital or other
- Study start
- 4 May 2022 (actual)
- Primary completion
- 16 Mar 2024 (actual)
- Study completion
- 16 Mar 2024 (actual)
- First posted
- 10 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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:
- 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:
- 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 design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Prevention (metformin) | Experimental | Drug: Metformin, Procedure: BiopsyPatients receive metformin PO QD on days 1-3 and then PO BID for up to 12 months in the absence of unacceptable toxicity. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Age 18-85
- Clinical appearance of lesion
- Histologic appearance
- 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
Key exclusion criteria (7 listed)
- 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
- Immunosuppression by natural illness or medically induced
- Hypersensitivity or allergic reaction to metformin or some other contraindication
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MetforminAlso: 1-Dimethylbiguanide, 657-24-9, N-dimethylbiguanide, N-Dimethylimidodicarbonimidic DiamideGiven PO | Drug | Prevention (metformin) |
| BiopsyAlso: BIOPSY_TYPE, BxUndergo biopsy | Procedure or surgery | Prevention (metformin) |
Primary outcome
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.
1 secondary outcome
Change in lesion status
Time frame: Baseline to 1 year after treatment
Results and safety
The study was stopped early (reason given: Slow accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Metformin and Biopsy in people with Erythroplakia and Leukoplakia, run by Thomas Jefferson University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Slow accrual). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Transformation-free-survival, in lesion types erythroplakia and verrucous hyperplasia (time frame: Up to 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 May 2025.