Study of metformin for cancer prevention in adolescents and adults with Li-Fraumeni syndrome

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What is this study about?

This study focuses on Li-Fraumeni syndrome (LFS), a rare inherited condition that increases the risk of developing various types of cancer. The research aims to determine if the medication metformin, commonly used to treat diabetes, can help prevent cancer in people with LFS when combined with regular cancer monitoring.

The study will evaluate two different forms of metformin: oral solution and film-coated tablets. Participants will receive a daily dose of up to 2000 milligrams of metformin for approximately 84 days. Some participants will receive metformin along with regular cancer surveillance, while others will only undergo cancer surveillance without receiving the medication.

During the study, participants will be monitored for any new cancer developments and their overall health status. The research team will track various factors, including the participants’ quality of life, emotional well-being, and any side effects from the treatment. They will also examine how factors like body weight and lifestyle choices might influence the treatment’s effectiveness.

1 Initial enrollment

After confirmation of Li-Fraumeni syndrome (LFS) diagnosis through genetic testing, you will be randomly assigned to one of two groups

The study will compare two approaches: close cancer surveillance with metformin treatment versus surveillance alone

2 Treatment assignment

If assigned to the metformin group, you will receive daily oral metformin in addition to regular cancer monitoring

If assigned to the control group, you will receive only regular cancer monitoring without medication

3 Monitoring period

Regular health assessments will track any cancer development

Medical staff will monitor for any side effects of the treatment

You will complete questionnaires about your quality of life and emotional well-being

Your weight and body mass index (BMI) will be measured

If you are in the metformin group, your medication adherence will be tracked

4 Long-term follow-up

The study continues until August 2032

Medical staff will document any cancer diagnoses, including details about size and type

Regular assessments will continue throughout the study period

Who Can Join the Study?

You must be at least 10 years old to participate
You can be of any gender (male, female, or diverse)
You must have confirmed Li-Fraumeni syndrome through genetic testing showing changes in the TP53 gene (either present from birth or developed later)
You must be able to understand the study process and what it means to participate in the trial
You must provide written consent to participate in the study
If you are under legal age, both of your parents or legal representatives must also provide written consent

Who Cannot Join the Study?

Age below 10 years old or above 65 years old
Known allergic reaction or sensitivity to metformin
Current pregnancy or planning to become pregnant during the study
Active cancer treatment at the time of enrollment
Severe kidney problems (as metformin is cleared by the kidneys)
Severe liver dysfunction
History of lactic acidosis (a condition where lactic acid builds up in the bloodstream)
Uncontrolled diabetes mellitus (high blood sugar levels)
Participation in other clinical trials within the past 30 days
Inability to take oral medication
Serious heart conditions or heart failure
Conditions that may interfere with regular study visits and follow-up
Mental health conditions that could affect ability to provide informed consent
Alcohol dependency or current substance abuse
Use of medications that may interact with metformin

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Medizinische Hochschule Hannover	Hanover	Germany
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country	Status
Uifcbwblsiukvcnrjwfnm Ajnprdfw	Augsburg	Germany

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	25.08.2025

Trial locations

Investigated drugs:

Metformin is a commonly prescribed medication that helps control blood sugar levels and is typically used to treat type 2 diabetes. In this trial, researchers are studying its potential new use for cancer prevention. The medication is being investigated for its ability to help prevent cancer in people with Li-Fraumeni syndrome (LFS), a rare genetic condition that increases the risk of developing various types of cancer. Patients take metformin daily in addition to their regular cancer surveillance program.

Li-Fraumeni syndrome – A rare inherited genetic condition caused by mutations in the TP53 gene, which normally helps prevent cancer formation. People with this syndrome have a significantly higher risk of developing various types of cancer throughout their lifetime. The condition can affect multiple generations within a family and typically appears in childhood or early adulthood. The syndrome can cause different types of cancers to develop in various parts of the body, including soft tissues, bones, breast, brain, and other organs. Individuals with Li-Fraumeni syndrome may develop multiple primary cancers over their lifetime.

Trial ID:

2025-520714-63-00

Protocol code:

LFS-Metformin-Trial

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of metformin for cancer prevention in adolescents and adults with Li-Fraumeni syndrome

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?