What side effects are associated with this type of intervention?

Common treatments for oral cancers include surgery, radiation therapy, and chemotherapy. Surgery can lead to side effects such as pain, swelling, and difficulty swallowing. Radiation therapy often causes mucositis, xerostomia (dry mouth), and taste changes. Chemotherapy can result in nausea, vomiting, hair loss, and increased risk of infections. Metformin Hydrochloride, being studied for chemoprevention, is generally well-tolerated but can cause gastrointestinal issues like nausea and diarrhea. It is important to discuss potential side effects with a healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

FDA-approved treatments for oral cancers primarily include therapeutic agents rather than chemopreventive ones. Commonly approved drugs for treating oral cancers include cetuximab, pembrolizumab, and nivolumab, which are used for their efficacy in targeting cancer cells. These drugs are typically used in cases of recurrent or metastatic squamous cell carcinoma of the head and neck. While Metformin Hydrochloride is being studied for its potential in chemoprevention, it is not yet FDA-approved for this purpose. Current FDA-approved treatments focus more on direct cancer therapy rather than prevention.

What other types of research exist?

Promising novel alternatives to Metformin Hydrochloride for chemoprevention in oral cancer patients include parathyroid hormone (teriparatide), pentoxifylline and vitamin E, low-level laser irradiation, hyperbaric oxygen, and topical application of medical ozone. These alternatives are still under investigation and have shown potential benefits in limited data, but none are yet considered standard approaches.

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Anti-diabetic drug

Metformin for Preventing Oral Cancer

Phase 2

Waitlist Available

Led By Scott M Lippman

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Karnofsky performance status >= 70%

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

Must not have

Patients with diabetes who are taking insulin or oral agents

History of allergic reactions attributed to compounds of similar chemical composition to metformin or prior use of metformin within the last year

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 14 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial tests if metformin hydrochloride can prevent oral cancer in patients with specific mouth lesions. These lesions can increase cancer risk, and metformin may help by slowing cell growth and affecting proteins related to cancer development. Metformin, widely used as a diabetes medication, has recently been reported to reduce cancer risk and improve outcomes in certain cancers.

Who is the study for?

This trial is for people with certain mouth lesions that could turn into cancer, specifically oral leukoplakia or erythroplakia. Participants should have a life expectancy over 3 months, be able to take pills, and not have diabetes treated with insulin or other drugs. They must also agree to use birth control and not have had any recent treatments for other cancers.

What is being tested?

The study is testing if Metformin Hydrochloride can prevent mouth cancer in patients with premalignant lesions. It's a phase IIa trial where the effects of the drug will be monitored through lab tests to see if it stops these lesions from becoming cancerous.

What are the potential side effects?

Metformin may cause side effects like stomach upset, diarrhea, nausea and vomiting, muscle pain, breathing difficulties, low vitamin B12 levels, and possibly lactic acidosis which is rare but serious.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am able to care for myself but may not be able to do active work.

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I have oral leukoplakia or erythroplakia with changes in the tissue, not caused by dentures.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have diabetes and am taking insulin or pills for it.

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I have had allergic reactions to metformin or similar drugs, or I've used metformin in the last year.

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I have liver disease, hepatitis, cirrhosis, or portal hypertension.

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I've had cancer treatment in the last 2 years, but ongoing hormone therapy for breast cancer is okay.

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I have a history of kidney disease.

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I do not have any uncontrolled illnesses that could affect my participation.

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I have a non-invasive mouth cancer.

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I have had diabetic ketoacidosis in the past.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 14 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 14 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 14 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Clinical Response to Metformin Intervention

Secondary study objectives

Change in Measurements of Metformin Hydrochloride Concentrations in Serum and Saliva

Change in Serum Metabolic Markers

Change in Serum and Saliva Inflammatory and Angiogenic Cytokines

+4 more

Other study objectives

Change in Saliva Microbiome Analyzed Using Flow Cytometry

Microbiome Signatures Correlated With Treatment Response

Side effects data

From 2023 Phase 2 trial • 40 Patients • NCT03733132

35%

Diarrhea

10%

Anemia

10%

Nausea

10%

Gastroesophageal reflux disease

Hepatobiliary disorders

Fall

Weight loss

COVID-19 Infection

Flatulence

Pain

Social circumstances

Vomiting

Headache

Stroke

Hypertension

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Metformin

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Prevention (extended-release metformin hydrochloride)Experimental Treatment2 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Metformin Hydrochloride

2016

Completed Phase 4

~1990

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Metformin Hydrochloride, commonly used for diabetes, is being studied for its potential to prevent oral cancer by inhibiting cellular proliferation and inducing apoptosis in premalignant cells. This is particularly relevant for patients with oral premalignant lesions, as it may reduce the risk of progression to cancer. Other common treatments for oral cancers include chemotherapy, which works by killing rapidly dividing cancer cells; targeted therapy, which blocks specific molecules involved in cancer growth and spread; and immunotherapy, which enhances the body's immune response against cancer cells. Understanding these mechanisms helps oral cancer patients and their doctors choose the most effective treatment strategy based on the cancer's characteristics and the patient's overall health.