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Study on Metformin for Preventing Leukemia in Patients with Clonal Cytopenia and Low-Risk Myelodysplastic Syndromes

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What is this trial about?

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

This clinical trial is focused on studying two specific blood disorders: Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS), particularly those with very low and low risk. These conditions involve issues with blood cell production in the bone marrow, which can lead to a shortage of healthy blood cells. The study is exploring the use of Metformin Hydrochloride, a medication commonly used to treat diabetes, to see if it can help prevent the progression of these blood disorders into leukemia.

The purpose of the study is to assess the safety of metformin and how feasible it is to use this treatment in patients with CCUS and low-risk MDS. Participants in the study will take metformin in the form of a coated tablet, which is taken orally. The study will last for up to 52 weeks, during which time researchers will monitor the participants' health and any changes in their condition. The study will also look at how metformin might work to control the progression of these diseases.

Throughout the study, researchers will collect information on various health markers, such as changes in blood cell counts and other indicators of disease progression. This information will help determine if metformin could be a viable option for preventing leukemia in patients with CCUS and low-risk MDS. The findings from this study will inform the design of future research to further explore the potential benefits of metformin for these conditions.

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 visit

    Upon joining the study, you will have an initial visit. During this visit, your eligibility for the trial will be confirmed. This involves reviewing your medical history and conducting necessary tests to ensure you meet the study criteria.

    You will be asked to provide written informed consent, confirming your understanding and willingness to participate in the study.

  2. Step 2

    Medication administration

    You will begin taking the study medication, metformin hydrochloride, which is administered orally. The specific dosage and frequency will be determined by the study team based on your individual needs and responses.

    The duration of the medication administration will be up to 12 months, during which you will be monitored regularly to assess the medication's effects and any potential side effects.

  3. Step 3

    Regular follow-up visits

    Throughout the study, you will have regular follow-up visits. These visits are scheduled to monitor your health, assess the progress of the study, and make any necessary adjustments to your treatment plan.

    During these visits, various tests and assessments will be conducted, including blood tests and questionnaires about your health and well-being.

  4. Step 4

    Final assessment

    At the end of the study period, a final assessment will be conducted. This will involve a comprehensive evaluation of your health and the effects of the study medication.

    The results of this assessment will help determine the safety and feasibility of using metformin hydrochloride as a preventive treatment for leukemia in patients with CCUS and LR-MDS.

Who can join the trial?

7 criteria

  • Must be diagnosed with either:
    • Very low- or low-risk myelodysplastic syndromes (MDS): A type of blood disorder with a specific risk level.
    • Clonal cytopenia of undetermined significance (CCUS): A condition with certain genetic changes and low blood cell counts, not explained by other diseases.
    • For women, must be in menopause: This means being over 45 years old and not having had a menstrual period for at least 12 months, without other medical reasons.
    • Must be at least 18 years old.
    • Must provide written informed consent: Agreeing to participate in the study after understanding all the details.
    • Must be willing to follow all required parts of the study.
    • Must be able to swallow pills.

Who cannot join the trial?

4 criteria

  • Patients with **very low- and low-risk myelodysplastic syndromes (MDS)** cannot participate. MDS is a group of disorders caused by poorly formed or dysfunctional blood cells.
  • Patients with **clonal cytopenia of undetermined significance (CCUS)** cannot participate. CCUS is a condition where there is a reduction in the number of blood cells, but the cause is not clear.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups like children, pregnant women, or those unable to give consent.
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Investigated drugs

Metformin is a medication commonly used to treat type 2 diabetes. In this clinical trial, it is being studied for a different purpose: to see if it can help prevent leukemia in patients with certain blood conditions called clonal cytopenia of undetermined significance (CCUS) and lower-risk myelodysplastic syndromes (LR-MDS). Researchers are interested in understanding how metformin might slow down or control the progression of these conditions. The trial aims to ensure that using metformin is safe for these patients and to gather information that could help design a larger study in the future.

What is already known about the treatment

Metformin – Metformin is typically administered orally in the form of tablets. It is currently being studied for its potential use in preventing leukemia in patients with clonal cytopenia of undetermined significance (CCUS) and low-risk myelodysplastic syndromes (LR-MDS). The main therapeutic indication being explored is its ability to control disease progression in these conditions. At the molecular level, metformin works by activating an enzyme called AMP-activated protein kinase (AMPK), which helps regulate energy balance and can inhibit cell growth. It is classified as a biguanide, a type of medication commonly used to manage blood sugar levels in diabetes.

Investigated diseases

  • Myelodysplastic Syndromes (MDS), Very Low- and Low-Risk

    Myelodysplastic syndromes are a group of disorders caused by poorly formed or dysfunctional blood cells. In very low- and low-risk MDS, the bone marrow produces an insufficient number of healthy blood cells, leading to anemia, infections, or easy bleeding. The disease progresses slowly, with gradual worsening of blood cell production. Over time, the bone marrow may become more dysfunctional, but the risk of progression to acute leukemia is lower compared to higher-risk MDS. Patients may experience fatigue, weakness, and other symptoms related to low blood counts. The condition is often monitored over time to assess changes in blood cell levels.

  • Clonal Cytopenia of Undetermined Significance (CCUS)

    Clonal cytopenia of undetermined significance is a condition characterized by a reduction in blood cell counts due to the presence of a clone of blood cells with genetic mutations. Despite the presence of these mutations, the condition does not meet the criteria for a specific blood cancer. The progression of CCUS is generally slow, with stable or gradually declining blood cell counts. Patients may experience symptoms related to low blood counts, such as fatigue or increased susceptibility to infections. The condition is monitored to detect any changes that might indicate progression to a more serious disorder. CCUS is considered a precursor state to more severe hematological diseases.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-518436-37-01Estimated enrolment70 patientsSponsorRigshospitalet

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).