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

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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 trial?

6 criteria

  • 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 trial?

15 criteria

  • 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
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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.

What is already known about the treatment

Metformin - An oral medication commonly prescribed as tablets that is being studied for cancer prevention in patients with Li-Fraumeni syndrome, a rare genetic condition that increases cancer risk. Originally developed and widely used as a first-line treatment for type 2 diabetes, metformin works by reducing glucose production in the liver and improving the body's sensitivity to insulin, but research suggests it may also have anti-cancer properties through its effects on cellular metabolism and growth pathways. This medication belongs to the biguanide class of drugs and is currently being investigated in clinical trials for its potential role in cancer prevention, particularly in high-risk populations, while maintaining its well-established safety profile from decades of use in diabetes treatment.

Investigated diseases

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2025-520714-63-00Protocol codeLFS-Metformin-TrialEstimated enrolment330 patientsSponsorMedizinische Hochschule Hannover

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