Active not recruitingPHASE2INTERVENTIONAL

Metformin for the Prevention of Oral Cancer in Patients With Oral Leukoplakia or Erythroplakia

This clinical trial is looking into whether a medicine called metformin, which is often used for diabetes, could help prevent oral cancer. It's for people who have certain white or red patches in their mouth, known as oral leukoplakia or erythroplakia, which can sometimes turn into cancer. Researchers want to see if metformin can stop these mouth changes from getting worse. Participants will be split into two groups: one receiving metformin and the other a dummy pill (placebo). They'll take their assigned medicine daily for 24 weeks, and doctors will carefully check the patches to see if metformin has any effect. The study aims to understand if this existing drug could offer a new way to prevent oral cancer.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

University of Arizona

Enrolment target

Start

12 Jan 2023

Estimated completion

01 Dec 2026

Erythroplakia Oral Leukoplakia

What is this study about?

This study is a clinical trial exploring whether a medicine called metformin could help reduce the risk of oral cancer. Metformin is a drug that many people already take to manage their blood sugar, especially if they have type 2 diabetes. However, doctors are now investigating if it might have other benefits, specifically for people who are at a higher risk of developing oral cancer.

The trial is looking for people who have certain kinds of patches in their mouth – either white patches called oral leukoplakia or red patches called erythroplakia. These patches are important because, in some cases, they can be pre-cancerous, meaning they have changes that could, over time, develop into oral cancer. The main goal is to see if metformin can either shrink these patches or stop them from changing further in a way that might lead to cancer.

Researchers will carefully compare what happens to the patches in people taking metformin versus those taking a dummy pill (placebo). This will help them understand if metformin really has an effect and how it might work. If successful, this research could lead to a new way to help prevent oral cancer for people with these specific mouth conditions.

Key takeaways

This study investigates if metformin can prevent oral cancer in people with pre-cancerous mouth patches.
It's for people with oral leukoplakia or erythroplakia.
Participants will take either metformin or a dummy pill for 24 weeks.
Regular blood tests and small tissue samples (biopsies) will be taken.
The study aims to find a new way to prevent oral cancer.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you need to be at least 21 years old and have specific white (leukoplakia) or red (erythroplakia) patches inside your mouth. These patches must be active and located in certain high-risk areas, like the floor of your mouth or your tongue, and meet specific size requirements. You also need to be a current or former smoker.

Beyond your mouth condition, the study needs to make sure you are generally well enough to participate. This means your routine blood tests, like your white blood cells, red blood cells, and liver and kidney function, need to be within a healthy range. Your overall physical ability to carry out day-to-day activities should also be good.

If you're a woman who could become pregnant, or a man whose partner could become pregnant, you'll need to agree to use reliable birth control methods throughout the study. This is because the effects of metformin on an unborn baby are not fully known, even though existing research in animals suggests it's unlikely to cause harm.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 21 years old or older?
Do you have white (leukoplakia) or red (erythroplakia) patches in your mouth that your doctor has identified?
Are these patches in areas like the floor of your mouth or tongue, and are they a specific size?
Are you a current or past smoker?
Are your general health and blood test results within healthy ranges (e.g., kidney and liver function, blood counts)?
Are you willing to use effective contraception during the study if it applies to you?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be randomly assigned to one of two groups. One group will take extended-release metformin tablets by mouth once a day, and the other group will take a dummy pill (placebo) by mouth once a day. You won't know which one you're receiving, nor will your study doctor. This treatment will last for 24 weeks (about 6 months).

Throughout the study, you'll have regular visits where doctors will collect blood samples and take small tissue samples (biopsies) from your mouth patches. These will be done at the start of the study and again at the end of the 24-week treatment period. These samples help the researchers understand how the patches are responding to the medication or placebo. After you finish the 24-week treatment, you'll have a final follow-up check-in about three weeks later.

Potential risks and benefits

Taking part in this study could potentially offer a benefit if metformin proves effective in preventing your oral patches from worsening or developing into cancer. However, there's no guarantee, as the study is designed to find this out. You might experience side effects from metformin, which commonly include stomach upset, nausea, or diarrhoea, though other more serious side effects are rare. Taking a dummy pill carries no direct medical risks, though there might be a risk that your condition progresses while you are in the placebo group. You can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (10)

University of Arizona Cancer Center-North Campus
Verified postcode
Tucson, United States
UC San Diego Medical Center - Hillcrest
Verified postcode
San Diego, United States
Moffitt Cancer Center
Verified postcode
Tampa, United States
Emory University Hospital/Winship Cancer Institute
Verified postcode
Atlanta, United States
Louisiana State University
Verified postcode
Lafayette, United States
University of Michigan Comprehensive Cancer Center
Verified postcode
Ann Arbor, United States
University of Minnesota/Masonic Cancer Center
Verified postcode
Minneapolis, United States
NYU College of Dentistry
Verified postcode
New York, United States
British Columbia Cancer Agency
Verified postcode
Vancouver, Canada
Dalhousie University
Verified postcode
Halifax, Canada

Common questions

What are oral leukoplakia and erythroplakia?

These are white (leukoplakia) or red (erythroplakia) patches inside your mouth that can sometimes, but not always, turn into cancer over time. Doctors monitor them closely.

What is metformin?

Metformin is a common medicine typically used to treat type 2 diabetes by helping to control blood sugar levels. This study is exploring if it has other benefits, like preventing cancer.

What is a 'placebo'?

A placebo is a dummy pill that looks exactly like the study medicine but contains no active drug. It helps researchers fairly compare the effects of the active medicine.

Will I know if I'm getting metformin or the placebo?

No, neither you nor your study doctor will know which you are receiving. This is called 'blinding' and helps ensure the study results are as fair and accurate as possible.

How long does the study last?

The main treatment period involves taking the study medication or placebo for 24 weeks (about 6 months). There will also be some initial checks and a final follow-up around three weeks after you finish treatment.