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Study on the Safety and Effectiveness of Mebendazole and Low-Dose Cytarabine for Elderly Patients with Relapsed or Refractory Acute Myeloid Leukemia

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 clinical trial is focused on studying a type of blood cancer called acute myeloid leukemia (AML), specifically in elderly patients whose disease has returned or did not respond to previous treatments. The study will test a combination of two medications: mebendazole, which is commonly used to treat parasitic infections, and cytarabine, also known as Ara-C, which is a chemotherapy drug. The purpose of the study is to evaluate the safety and effectiveness of this combination in treating AML.

Participants in the study will receive the medications over a period of time, with the treatment being administered in cycles. The study will be conducted in two phases. The first phase will focus on determining a safe dose of mebendazole when used with low-dose Ara-C. The second phase will assess how well the treatment works in achieving remission, which means reducing or eliminating signs of cancer in the body. The study aims to find out if this combination can help improve the condition of patients with relapsed or refractory AML.

Throughout the study, participants will be monitored for any side effects and changes in their health. The researchers will also look at how the treatment affects the cancer cells and the overall survival of the patients. This study is important for finding new ways to treat AML in patients who are not eligible for more intensive treatments. The results could provide valuable information for developing better treatment options for this challenging condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria such as age, diagnosis, and ability to take oral medication.

    Informed consent is required, ensuring understanding of the study's purpose and procedures.

  2. Step 2

    Phase I treatment

    The initial phase focuses on evaluating the safety of the treatment combination.

    Participants receive mebendazole and low-dose Ara-C (cytarabine).

    Mebendazole is administered orally in tablet form, with a dosage of either 500 mg or 100 mg, depending on the specific protocol.

    Ara-C is administered subcutaneously, meaning it is injected under the skin.

    The goal is to determine a safe and effective dose of mebendazole in combination with Ara-C.

  3. Step 3

    Monitoring and assessment

    Throughout the treatment, the frequency and severity of any side effects are recorded.

    Regular assessments are conducted to monitor the body's response to the treatment.

    This includes checking blood counts and other health indicators.

  4. Step 4

    Phase II treatment

    The second phase aims to evaluate the efficacy of the treatment combination.

    The focus is on achieving complete remission or partial remission with incomplete blood count recovery within three months.

    Participants continue to receive the established dose of mebendazole and low-dose Ara-C.

  5. Step 5

    Outcome evaluation

    The study measures the rate of remission and overall survival.

    Additional tests may be conducted to assess the presence of any remaining disease.

  6. Step 6

    End of study

    The study is expected to conclude by June 2026.

    Final results will be analyzed to determine the overall success and safety of the treatment.

Who can join the trial?

6 criteria

  • Have a diagnosis of **acute myeloid leukemia (AML)**, which is a type of blood cancer, except for a specific type called **promyelocytic leukemia (AML M3)**.
  • The disease must have returned or not responded to standard treatment, which is known as **relapsed/refractory disease**.
  • Have a high number of cancer cells, called **blasts**, in the blood, specifically more than 1000 per microliter.
  • Be a man or woman older than 70 years and not able to undergo intensive treatment, which means the usual strong doses of medicine.
  • Be able to swallow and keep down oral medication, not have any known issues with absorbing nutrients, and have organs that are working well enough.
  • Be able to understand the study and agree to participate by signing a form, which is known as **providing informed consent**.

Who cannot join the trial?

4 criteria

  • Patients who are not diagnosed with relapsed/refractory acute myeloid leukemia cannot participate. This is a type of blood cancer that has returned or did not respond to treatment.
  • Patients under the age of 70 cannot participate. The study is for elderly patients aged 70 years or older.
  • Patients who are not able to safely take the study medications, which include low-dose Ara-C and mebendazole, cannot participate. Ara-C is a chemotherapy drug, and mebendazole is a medication usually used to treat worm infections.
  • Patients who are part of a vulnerable population, such as those who cannot give consent or are unable to understand the study, cannot participate.
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Investigated drugs

  • Mebendazole

    is a medication being studied for its potential use in treating elderly patients with relapsed or refractory acute myeloid leukemia (AML). In this trial, researchers are examining how safe and effective it is when used in combination with another medication. Mebendazole is traditionally used to treat parasitic worm infections, but in this study, its role is to help fight cancer cells in the blood.

  • Ara-C

    , also known as cytarabine, is a chemotherapy drug commonly used to treat certain types of leukemia, including AML. In this trial, a low dose of Ara-C is combined with mebendazole to see if the combination can improve treatment outcomes for elderly patients whose leukemia has returned or has not responded to previous treatments. The goal is to find a safe and effective dose that can help achieve remission in these patients.

What is already known about the treatment

Mebendazole – Mebendazole is typically administered orally in tablet form. It is currently being studied in clinical trials for its potential use in treating relapsed or refractory acute myeloid leukemia, particularly in elderly patients. The main therapeutic indication being explored is its combination with low-dose Ara-C to improve treatment outcomes in this patient group. At the molecular level, mebendazole works by disrupting the formation of microtubules, which are essential for cell division, thereby inhibiting the growth of cancer cells. It is classified pharmacologically as an anthelmintic, traditionally used to treat parasitic worm infections, but is now being investigated for its anti-cancer properties.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia (AML) is a type of cancer that starts in the blood-forming cells of the bone marrow and quickly progresses to affect the blood. It is characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. This leads to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. AML can occur at any age but is more common in older adults. The disease can be classified as relapsed or refractory when it does not respond to treatment or returns after a period of improvement. The progression of AML can vary, but it typically requires prompt medical attention to manage symptoms and complications.
Trial detailsLast updated 7 Oct 2026
Age65+ yearsPhasePhase IITrial ID2024-518363-36-00Protocol codeMAILEstimated enrolment50 patientsSponsorUniversity Medical Center Hamburg-Eppendorf

sourced from the EU Clinical Trials Register and site verification

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