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Study of Bleximenib, Venetoclax, and Azacitidine Treatment for Newly Diagnosed Acute Myeloid Leukemia Patients Ineligible 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 study focuses on Acute Myeloid Leukemia (AML), a type of cancer that affects the blood and bone marrow. The study specifically targets patients with newly diagnosed AML who have certain genetic changes called KMT2A rearrangements or NPM1 mutations and who cannot receive intensive chemotherapy. The purpose of this study is to determine if adding a medication called bleximenib to a combination of venetoclax and azacitidine (VEN+AZA) works better than using venetoclax and azacitidine alone.

The treatment involves taking these medications in cycles. Some participants will receive bleximenib along with venetoclax and azacitidine, while others will receive venetoclax and azacitidine with a placebo. This is a double-blind study, which means neither the participants nor the researchers will know which treatment group a participant is in until the study is completed.

The study will monitor how participants respond to treatment by tracking various health indicators and will assess whether the addition of bleximenib improves complete remission (when there are no detectable cancer cells) and overall survival in patients with this type of leukemia.

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

    Start of Trial

    After joining the study, you will be randomly assigned to one of two treatment groups. One group will receive bleximenib, venetoclax, and azacitidine, while the other group will receive venetoclax, azacitidine, and a placebo (an inactive substance that looks like bleximenib).

    This is a double-blind study, which means neither you nor your doctor will know which treatment group you are in.

  2. Step 2

    Treatment Administration

    Your treatment will consist of 28-day cycles of medication:

    Venetoclax (Venclyxto): You will take this medication in tablet form. Venetoclax comes as 50 mg and 100 mg film-coated tablets.

    Azacitidine: This medication will be given as an injection under your skin or through an intravenous (IV) line. The formulation used is Azacitidine betapharm 25 mg/mL powder for suspension for injection.

    Bleximenib or matching placebo: Depending on your assigned group, you will receive either bleximenib (30 mg, 50 mg, or 100 mg) or a matching placebo in the form of film-coated tablets.

  3. Step 3

    Monitoring and Assessments

    Throughout the trial, you will have regular visits to monitor your health and the effectiveness of the treatment.

    These visits will include blood tests to check your blood cell counts and assess how well your kidneys and liver are functioning.

    Bone marrow assessments will be performed periodically to evaluate how the leukemia is responding to treatment.

    Your doctor will also monitor you for any side effects or complications related to the treatment.

  4. Step 4

    Treatment Duration

    You will continue receiving treatment in 28-day cycles as long as you are benefiting from it and not experiencing unacceptable side effects.

    The study will track two main outcomes: complete remission (CR), which means no detectable cancer cells, and overall survival (OS), which is how long patients live after starting treatment.

  5. Step 5

    End of Treatment

    Your participation in the treatment phase will end if:

    Your disease progresses despite treatment

    You experience unacceptable side effects

    You decide to withdraw from the study

    Your doctor decides it's in your best interest to stop the study treatment

    The study itself is terminated

  6. Step 6

    Follow-up Period

    After completing or discontinuing treatment, you will enter a follow-up period.

    During this time, your health status will continue to be monitored through periodic check-ups and assessments.

    The entire study is expected to run until 2029, though your active participation may be shorter depending on your individual treatment course.

Who can join the trial?

10 criteria

  • You must be 18 years of age or older (or the legal age of majority in your location, whichever is greater) when you sign the informed consent.
  • You must have previously untreated acute myeloid leukemia (a type of blood cancer) with specific genetic mutations called KMT2Ar or NPM1m, with at least 10% blasts (immature blood cells) according to 2022 criteria.
  • You must not be eligible for intensive chemotherapy due to one of these reasons:
    • You are 75 years or older and your doctor determines you're not eligible, with an ECOG performance status (a measure of your ability to perform daily activities) of 0-2
    • You are between 18 and 74 years old with at least one of these health issues:
      • ECOG performance status of 2
      • Severe heart condition
      • Severe lung condition
      • Kidney impairment
      • Other health condition that makes you unsuitable for intensive chemotherapy
      • You must have adequate kidney and liver function before randomization, based on specific blood test results.
      • Your white blood cell (WBC) count must be less than 25 × 10^9/L (less than 25 billion white blood cells per liter of blood).

Who cannot join the trial?

11 criteria

  • You cannot participate if you are under 18 years old.
  • You cannot participate if you have received previous treatment for acute myeloid leukemia (a type of blood cancer affecting bone marrow cells).
  • You cannot participate if you have acute promyelocytic leukemia (a specific subtype of acute myeloid leukemia).
  • You cannot participate if you have an active central nervous system involvement with leukemia (cancer cells in the brain or spinal cord).
  • You cannot participate if you have another type of cancer that requires treatment during the study period.
  • You cannot participate if you have severe heart problems or uncontrolled high blood pressure.
  • You cannot participate if you have severe liver or kidney disease.
  • You cannot participate if you have an active, uncontrolled infection.
  • You cannot participate if you are pregnant or breastfeeding.
  • You cannot participate if you have had major surgery within 4 weeks before starting the study treatment.
  • You cannot participate if you are currently enrolled in another investigational drug study.
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Investigated drugs

  • Bleximenib

    is a medication being studied for the treatment of acute myeloid leukemia (AML). It is designed to target specific genetic changes in cancer cells, particularly in patients with KMT2A rearrangements or NPM1 mutations. This medication is being tested to see if it can improve outcomes when added to other treatments.

  • Venetoclax

    is a medication that works by blocking a protein called BCL-2, which helps cancer cells survive. By blocking this protein, venetoclax can help trigger cancer cells to die. It is commonly used in combination with other medications to treat various blood cancers, including acute myeloid leukemia.

  • Azacitidine

    is a medication that affects how genes are expressed in cells. It helps bone marrow produce normal blood cells and kills abnormal cells in the bone marrow. Azacitidine is commonly used to treat certain types of blood disorders and cancers, including acute myeloid leukemia, particularly in patients who cannot tolerate intensive chemotherapy.

What is already known about the treatment

  • Bleximenib

    An investigational oral medication currently being studied in clinical trials for acute myeloid leukemia (AML), particularly in patients with specific genetic mutations (KMT2A rearrangements or NPM1 mutations). It works at the molecular level by targeting specific cancer cell mechanisms, though its exact mechanism of action is still being fully characterized in ongoing research. Bleximenib belongs to a class of novel targeted therapies being developed to improve outcomes in AML patients who cannot receive intensive chemotherapy. It is being evaluated in combination with venetoclax and azacitidine to determine if this combination provides better efficacy compared to standard treatment approaches.

  • Venetoclax

    An oral medication approved for treating certain blood cancers that works by blocking the BCL-2 protein, which helps cancer cells survive. It functions by promoting cancer cell death (apoptosis) by preventing BCL-2 from protecting leukemia cells from natural death processes. Venetoclax is classified as a BCL-2 inhibitor and is currently used in combination with other medications for treating acute myeloid leukemia in patients who cannot receive intensive chemotherapy due to age or other health factors. In clinical practice, it has demonstrated significant effectiveness when combined with hypomethylating agents like azacitidine, making it an important component of less-intensive treatment regimens for AML.

  • Azacitidine

    A medication administered by injection that is used to treat certain types of blood cancers including acute myeloid leukemia. It works by interfering with DNA, helping to restore normal function to genes that control cell growth and differentiation, potentially allowing bone marrow to produce normal blood cells. Azacitidine is classified as a hypomethylating agent or DNA demethylating agent and is commonly used in patients who cannot tolerate more intensive chemotherapy. It has become a standard treatment option for older adults with AML and is often combined with other medications like venetoclax to improve treatment outcomes.

Investigated diseases

Acute Myeloid Leukemia - A rapidly developing cancer that originates in the bone marrow from myeloid stem cells that should normally develop into mature blood cells. In this disease, abnormal white blood cells accumulate in the bone marrow and interfere with the production of normal blood cells. As the disease progresses, these abnormal cells can spread to the bloodstream and potentially to other parts of the body including the lymph nodes, spleen, liver, and central nervous system. The disease typically develops quickly, over days or weeks, causing symptoms such as fatigue, shortness of breath, easy bruising and bleeding, and increased risk of infection. Without the proper production of healthy blood cells, individuals with this condition experience decreasing levels of red blood cells, platelets, and normal white blood cells.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-520154-38-00Protocol code75276617AML3001Estimated enrolment584 patientsSponsorJanssen Cilag International

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