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Study on the Effectiveness of Decitabine and Venetoclax for Patients with Acute Myeloid Leukemia Unfit for Intensive Chemotherapy

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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 treatment for patients with Acute Myeloid Leukemia (AML) that develops from a group of blood disorders known as myeloproliferative neoplasms. These patients are not suitable for intensive chemotherapy. The study is testing a combination of two medications: decitabine and venetoclax. Decitabine is given as a solution through a vein, while venetoclax is taken as a tablet by mouth.

The purpose of the study is to evaluate how effective this combination treatment is in improving the condition of patients with this type of leukemia. Participants will receive the treatment over a period of time, and their health will be monitored to see how well the treatment works and to check for any side effects. The study will also look at how long patients live without the disease getting worse and how the treatment affects their overall survival.

Throughout the study, researchers will collect information on how patients respond to the treatment, including any changes in their blood counts and overall health. The study aims to provide valuable insights into the potential benefits of using decitabine and venetoclax together for treating this specific type of leukemia. Participants will be closely monitored by healthcare professionals to ensure their safety and well-being during the trial.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and current health status.

    Eligibility criteria include a diagnosis of acute myeloid leukemia secondary to myeloproliferative neoplasms, being 60 years or older, or being unfit for intensive treatment.

  2. Step 2

    Treatment initiation

    The treatment involves a combination of two medications: decitabine and venetoclax.

    Decitabine is administered as an intravenous infusion. The dosage and frequency are determined by the healthcare provider based on individual needs.

    Venetoclax is taken orally in the form of film-coated tablets. The dosage may vary, with available strengths of 10 mg, 50 mg, and 100 mg.

  3. Step 3

    Treatment cycles

    The treatment is administered in cycles. Each cycle's duration and the number of cycles depend on the patient's response to the treatment.

    The primary goal is to achieve event-free survival, which means the absence of disease progression or relapse.

  4. Step 4

    Monitoring and evaluation

    Regular monitoring is conducted to assess the treatment's effectiveness and any side effects. This includes blood tests and other necessary evaluations.

    The response to treatment is evaluated at specific time points after each cycle to determine the overall response rate and disease status.

  5. Step 5

    End of treatment

    The treatment concludes based on the healthcare provider's assessment of the patient's condition and response to the therapy.

    Post-treatment follow-up may be required to monitor long-term outcomes and any potential late effects of the treatment.

Who can join the trial?

5 criteria

  • Patients must have a type of blood cancer called Acute Myeloid Leukemia (AML) that developed from another condition known as myeloproliferative neoplasms (MPN). This should be newly diagnosed and untreated, based on specific medical criteria.
  • Patients should be 60 years or older, or adults who are not suitable for intensive treatment, as decided by the doctor.
  • Patients should have an ECOG performance status of 0 to 2. This is a scale that measures how well a patient can perform daily activities. If the score is 3 due to the disease, it should be reversible with proper care.
  • Patients must sign a written consent form, agreeing to participate in the study, following international and local laws.
  • Male patients with partners who can become pregnant must agree to use a reliable method of birth control during the study. They should continue using it for 3 months after the last dose of a drug called decitabine. Female partners should use birth control for 1 month after the last dose of another drug called venetoclax, or 6 months after the last dose of decitabine, whichever is longer.

Who cannot join the trial?

5 criteria

  • Patients who have a different type of cancer that is not related to Acute Myeloid Leukemia (AML) secondary to myeloproliferative neoplasms (MPN). MPN is a group of diseases where the bone marrow makes too many blood cells.
  • Patients who are fit for intensive chemotherapy. Intensive chemotherapy is a strong treatment that uses drugs to kill cancer cells.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the specified clinical trial group.
  • Patients who are not considered part of the vulnerable population selected for the study.
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Investigated drugs

  • Decitabine

    is a medication used in this trial to help treat patients with acute myeloid leukemia (AML) that has developed from myeloproliferative neoplasms. It works by interfering with the growth of cancer cells, which may slow down or stop their spread.

  • Venetoclax

    is another medication included in the trial. It is designed to help treat AML by targeting and blocking a specific protein that cancer cells need to survive. This can lead to the death of the cancer cells and potentially improve patient outcomes.

What is already known about the treatment

  • Decitabine

    Decitabine is administered intravenously, typically in a hospital setting. It is currently used in medical practice and literature for treating certain types of blood cancers, including acute myeloid leukemia (AML). The main therapeutic indication for decitabine is to help manage AML, especially in patients who are not suitable for intensive chemotherapy. At the molecular level, decitabine works by incorporating into DNA and inhibiting DNA methyltransferase, leading to hypomethylation and reactivation of silenced genes that control cell growth. It is classified as a hypomethylating agent.

  • Venetoclax

    Venetoclax is taken orally in the form of tablets. It is widely recognized in medical literature and practice for its role in treating certain blood cancers, including AML. The primary therapeutic use of venetoclax is to target and kill cancer cells in patients with AML, particularly those who cannot undergo intensive chemotherapy. Venetoclax works by inhibiting the BCL-2 protein, which helps cancer cells survive, thereby promoting their death. It is classified as a BCL-2 inhibitor.

Investigated diseases

Acute Myeloid Leukemia secondary to myeloproliferative neoplasms – This is a type of cancer that starts in the blood-forming cells of the bone marrow and progresses rapidly. It occurs as a secondary condition following myeloproliferative neoplasms, which are disorders characterized by the overproduction of blood cells. In this disease, immature white blood cells, known as myeloblasts, accumulate in the bone marrow and interfere with the production of normal blood cells. As the disease progresses, it can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding due to the lack of healthy blood cells. The progression of the disease can vary, but it typically involves a rapid increase in the number of abnormal cells, which can spread to other parts of the body.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-510241-16-00Protocol codeAML2420Estimated enrolment101 patientsSponsorFondazione Gimema Franco Mandelli Onlus

sourced from the EU Clinical Trials Register and site verification

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