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Study of Bleximenib with Drug Combination for Patients with Acute Myeloid Leukemia with KMT2A or NPM1 Alterations

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). The study is particularly interested in cases of AML that have specific genetic changes known as KMT2A or NPM1 alterations. The trial will test a new treatment called Bleximenib, which will be used in combination with other therapies specifically directed at treating AML. Bleximenib is being tested to find the best dose and to see how safe and tolerable it is when used with these other treatments.

Participants in the study will receive Bleximenib along with other AML-directed therapies. The study will be conducted in two main parts. The first part will focus on determining the best dose of Bleximenib to use. The second part will further evaluate the safety and tolerability of this combination treatment. The study will involve regular monitoring to check for any side effects and to ensure the safety of the participants.

The purpose of this study is to explore the potential benefits of Bleximenib in treating AML with specific genetic changes. The study will help researchers understand how well Bleximenib works in combination with other treatments and what side effects might occur. This information could be valuable in developing new treatment options for people with AML in the future.

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 a physical examination.

    Blood tests are performed to ensure white blood cell count is less than or equal to 25 x 10^9 per liter, and liver and kidney functions are adequate.

  2. Step 2

    Consent and preparation

    A consent form is signed to confirm understanding of the study's purpose and procedures.

    For females of childbearing potential, a pregnancy test is conducted within 48 hours before the first dose of treatment.

  3. Step 3

    Treatment phase 1

    The first phase involves determining the recommended dose of bleximenib in combination with other therapies for acute myeloid leukemia (AML).

    Medications include bleximenib (oral use), cytarabine (intravenous use), venetoclax (oral use), idarubicin (intravenous use), and azacitidine (injection).

    The frequency and duration of administration depend on the specific combination and response to treatment.

  4. Step 4

    Treatment phase 2

    The second phase focuses on further evaluating the safety and tolerability of the selected dose of bleximenib in combination with AML therapies.

    Regular monitoring is conducted to assess the incidence and severity of any adverse effects.

  5. Step 5

    Follow-up and monitoring

    Throughout the trial, regular follow-up visits are scheduled to monitor health status and response to treatment.

    Blood tests and other assessments are conducted to ensure safety and effectiveness of the treatment.

Who can join the trial?

14 criteria

  • Must have a diagnosis of Acute Myeloid Leukemia (AML) according to World Health Organization (WHO) criteria. This includes:
    • De novo AML, which means it is a new diagnosis.
    • Secondary AML, which means it developed from a previous blood disorder.
    • Relapsed/Refractory AML (for Arm A only), which means the leukemia has returned or is not responding to treatment.
    • Must have specific genetic changes: NPM1, KMT2A, NUP98, or NUP214 alterations.
    • Must have certain blood test results:
      • White blood cell (WBC) count must be less than or equal to 25 x 109 per liter (L).
      • Must have adequate liver and kidney function.
      • Must have an ECOG performance status of 0, 1, or 2. This is a scale used to assess how well a person can perform daily activities:
        • 0 means fully active.
        • 1 means restricted in physically strenuous activity but can do light work.
        • 2 means capable of all self-care but unable to carry out any work activities.
        • If female and able to have children, must have a negative pregnancy test before starting the study.
        • Must sign an informed consent form (ICF), which means you understand the study and agree to participate.
        • Must be willing and able to follow the rules and restrictions of the study.
        • Open to both males and females.
        • Participants must be within certain age ranges, which typically include adults and older adults.

Who cannot join the trial?

10 criteria

  • Patients with any other type of cancer besides Acute Myeloid Leukemia (AML) cannot participate.
  • Patients who have had a heart attack or stroke in the past 6 months are not eligible.
  • Patients with uncontrolled high blood pressure cannot join the study.
  • Patients with severe liver disease are excluded.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients with active infections that require treatment are not eligible.
  • Patients who have received another investigational drug within the last 30 days cannot join.
  • Patients with known allergies to the study drug or its components are excluded.
  • Patients with a history of drug or alcohol abuse in the past year are not eligible.
  • Patients who are unable to comply with the study procedures cannot participate.
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Investigated drugs

Bleximenib is a medication being studied for its potential to help treat a type of blood cancer called Acute Myeloid Leukemia (AML). This medication is being tested in combination with other treatments specifically aimed at AML. The goal is to find out how well it works and how safe it is for patients who have certain genetic changes in their cancer, known as KMT2A or NPM1 alterations. Researchers are trying to determine the best dose to use and to see how patients respond to this combination of treatments.

What is already known about the treatment

Bleximenib – Bleximenib is administered orally and is currently being studied in clinical trials for its potential use in treating Acute Myeloid Leukemia (AML), particularly in patients with specific genetic alterations known as KMT2A or NPM1. This medication is still under investigation, and its safety and effectiveness are being evaluated in combination with other AML-directed therapies. Bleximenib works by targeting specific proteins involved in the growth and survival of cancer cells, aiming to stop or slow down the progression of the disease. It is classified as a targeted therapy, which means it specifically aims at cancer cells with certain characteristics, potentially leading to fewer side effects compared to traditional chemotherapy.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia is a type of cancer that starts in the blood-forming cells of the bone marrow. It progresses rapidly, leading to the accumulation of immature white blood cells called myeloblasts. These abnormal cells crowd out normal blood cells, causing symptoms like fatigue, frequent infections, and easy bruising or bleeding. As the disease advances, it can spread to other parts of the body, including the lymph nodes, liver, and spleen. The rapid growth of these abnormal cells can interfere with the production of normal blood cells. This disruption in blood cell production can lead to anemia, increased risk of infections, and bleeding problems.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-506582-58-00Protocol code75276617ALE1002Estimated enrolment146 patientsSponsorJanssen - Cilag International

sourced from the EU Clinical Trials Register and site verification

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