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Study of Ziftomenib (KO-539) in Patients with Relapsed or Refractory Acute Myeloid Leukemia

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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 patients with Acute Myeloid Leukemia (AML) that has come back or is not responding to previous treatments. The research evaluates a new medication called ziftomenib (also known as KO-539), which works by blocking a protein called menin that plays a role in the development of leukemia. The medication comes in the form of hard capsules that are taken by mouth.

The purpose of this research is to test how well ziftomenib works in treating people with AML and to find the most appropriate dose that can be given safely. The study is divided into different parts, with the first part focusing on finding the right dose and checking the safety of the medication. The later parts of the study look at how effective the treatment is for different groups of patients with specific genetic changes in their leukemia cells.

During the study, participants receive ziftomenib and are monitored regularly to check how they respond to the treatment. The researchers will collect blood samples and perform various tests to evaluate how well the medication is working and to check for any side effects. The study will also look at how long the beneficial effects of the treatment last and how it affects patients' overall survival.

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 evaluation

    Your eligibility for the study will be assessed based on the following criteria:

    You must be 18 years or older with relapsed or refractory acute myeloid leukemia (AML)

    Your bone marrow must show at least 5% cancer cells

    Your white blood cell count must be 30,000/μL or less

    Your physical condition score (ECOG) must be 2 or better, with a life expectancy of at least 2 months

  2. Step 2

    Treatment administration

    You will receive ziftomenib in the form of hard capsules

    The medication will be taken by mouth

    The exact dose will be determined during the study

    Blood samples will be collected at specific times to measure drug levels in your body

  3. Step 3

    Monitoring and assessment

    Your safety and response to treatment will be monitored throughout the study

    Regular evaluations will include:

    Blood tests to check your health status

    Assessment of any side effects

    Monitoring of your physical condition

    Evaluation of how well the treatment is working

  4. Step 4

    Study duration

    The study is scheduled to run from August 2021 to January 2029

    Your individual participation duration will depend on how you respond to the treatment

Who can join the trial?

7 criteria

  • Must be aged 18 years or older
  • Must have relapsed or refractory acute myeloid leukemia (AML) - this means the disease has either returned after treatment or did not respond adequately to standard treatments
  • Must have failed or be unsuitable for standard treatments, including stem cell transplant (a procedure where damaged bone marrow is replaced with healthy cells)
  • Must have at least 5% blast cells in bone marrow (blast cells are immature blood cells that indicate presence of leukemia)
  • Must have a white blood cell count of 30,000/μL or less in the blood
  • Must have an ECOG performance status of 2 or better (this measures how well you can perform daily activities and care for yourself)
  • Must have a life expectancy of at least 2 months

Who cannot join the trial?

17 criteria

  • Patients younger than 18 years old cannot participate
  • Prior history of severe allergic reactions to similar medications
  • Active uncontrolled infections requiring intravenous antibiotics
  • Presence of central nervous system leukemia (cancer cells in the brain or spinal fluid)
  • Pregnant or breastfeeding women
  • Severe heart conditions, including:
    • Uncontrolled high blood pressure
    • Recent heart attack (within 6 months)
    • Severe heart rhythm problems
    • Severe liver problems (liver function tests more than 3 times the normal limit)
    • Severe kidney problems (requiring dialysis)
    • Other active cancers requiring treatment
    • Major surgery within 4 weeks before starting the study
    • Participation in other clinical trials within 30 days before this study
    • Mental conditions that could interfere with following study procedures
    • Life expectancy less than 3 months
    • HIV (Human Immunodeficiency Virus) positive patients
    • Active hepatitis B or C infection
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Investigated drugs

Ziftomenib (also known as KO-539) is a Menin-MLL (KMT2A) inhibitor medication. It is being studied for treating patients who have acute myeloid leukemia (AML) that has either come back after previous treatment or has not responded to treatment. This medication works by targeting specific genetic changes in leukemia cells, particularly in patients who have certain mutations like NPM1 or KMT2A rearrangements. It is also being studied in patients who have acute lymphoblastic leukemia (ALL) with specific genetic changes.

What is already known about the treatment

Ziftomenib - An investigational oral medication being studied for the treatment of relapsed or refractory Acute Myeloid Leukemia (AML), specifically targeting patients with NPM1 mutations and KMT2A rearrangements. This drug works by inhibiting the interaction between Menin and MLL (KMT2A) proteins at the molecular level, which plays a crucial role in leukemia development. Currently undergoing Phase 1/2 clinical trials, ziftomenib is being evaluated for its safety, optimal dosing, and therapeutic effectiveness in patients whose leukemia has returned or become resistant to previous treatments. The medication belongs to the class of targeted cancer therapies known as Menin-MLL inhibitors.

Investigated diseases

  • Acute Myeloid Leukemia (AML)

    A blood and bone marrow cancer that affects the myeloid cells, which normally develop into various types of blood cells. In AML, the bone marrow produces abnormal white blood cells that do not function properly and multiply rapidly. These abnormal cells build up in the bone marrow and blood, leaving less room for healthy blood cells. The disease can progress quickly, causing a decrease in normal blood cell production, including red blood cells, white blood cells, and platelets. AML can occur in both children and adults, though it is more common in older adults. The condition may develop as a new disease or can emerge from other blood disorders.

  • Acute Lymphoblastic Leukemia (ALL)

    A blood cancer that affects lymphoid cells, which normally develop into white blood cells called lymphocytes. In ALL, immature lymphoid cells multiply rapidly and abnormally in the bone marrow, preventing the production of normal blood cells. These abnormal cells, called lymphoblasts, crowd the bone marrow and can spread to other parts of the body. The disease typically develops quickly, affecting the body's ability to produce normal blood cells. ALL is more common in children but can occur at any age.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2023-510509-17-00Protocol codeKO-MEN-001Estimated enrolment205 patientsSponsorKura Oncology Inc.

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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).