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A Study of Gilteritinib to Eliminate Remaining Cancer Cells in Patients with Acute Myeloid Leukemia and FLT3-ITD Mutation

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study involves patients with acute myeloid leukemia, a type of blood cancer that affects the bone marrow and blood cells. Specifically, the study focuses on patients whose cancer cells have a particular genetic change called FLT3-ITD mutation, which means the cancer cells have an abnormal version of a specific gene. The treatment being tested is gilteritinib, which is also known by its brand name Xospata. This medication belongs to a group of drugs called tyrosine kinase inhibitors, which work by blocking certain proteins that help cancer cells grow and survive. The study will look at patients who still have small amounts of cancer cells remaining in their body, called measurable residual disease, even after they have already received two rounds of intensive chemotherapy combined with another similar medication.

The purpose of the study is to see how well gilteritinib works at eliminating these remaining cancer cells that can be detected using a special laboratory test called PCR-NGS. During the study, patients will receive gilteritinib tablets by mouth for up to eight months. The medication will be given at a dose of up to 120 milligrams per day. Throughout the treatment period, doctors will monitor patients regularly to check if the remaining cancer cells have disappeared and to observe how patients respond to the medication.

The study will also examine several additional aspects, including what types of genetic changes might appear if the cancer comes back, how long the treatment works before any resistance develops, and whether certain characteristics of the cancer cells or the patient's immune system can predict how well the treatment will work. Doctors will use various laboratory tests to analyze changes in cancer cells and to look for specific genetic markers that might be related to treatment response. The study will also investigate whether certain mutations present alongside the FLT3-ITD mutation, such as those in genes called DNMT3A, NPM1, TET2, ASXL1, and TP53, have any effect on how patients respond to treatment.

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

    Signing informed consent and initial assessments

    Before starting the study, you will need to sign and date an informed consent form that has been approved by the appropriate ethics committees.

    You will undergo screening procedures to confirm your eligibility for the study.

    If you are a female of childbearing potential, you will have a serum pregnancy test during the screening visit and a urine pregnancy test within seven days before starting treatment with gilteritinib.

    Your performance status will be assessed using the ECOG scale, which measures how the disease affects your daily living abilities. Your score must be less than 2 to participate.

    Your measurable residual disease, also called MRD, will be tested using a laboratory technique called PCR-NGS to detect the FLT3-ITD mutation. This test looks for very small amounts of cancer cells that may remain in your body after chemotherapy.

  2. Step 2

    Beginning treatment with gilteritinib

    You will begin taking gilteritinib, which is provided as 40 mg film-coated tablets that are taken orally (by mouth).

    The treatment is administered in cycles. Each cycle represents a specific period of treatment.

    You will receive gilteritinib after having completed 2 cycles of intensive chemotherapy combined with an FLT3 inhibitor and after testing positive for MRD.

    The exact dosage, frequency, and duration of gilteritinib administration will be determined by the study protocol and your treating physician.

  3. Step 3

    First evaluation after 2 cycles of gilteritinib treatment

    After completing 2 cycles of treatment with gilteritinib, your MRD status will be evaluated again using the PCR-NGS test for FLT3-ITD.

    This test will determine whether the measurable residual disease has been eliminated from your body.

    Additional laboratory tests will be performed to assess your response to treatment.

  4. Step 4

    Ongoing monitoring and follow-up visits

    You will need to return to the clinical site for regular follow-up visits as required by the study protocol.

    During these visits, your health status will be monitored and any side effects will be assessed.

    Blood samples and bone marrow samples may be collected to evaluate various markers, including genetic mutations, changes in cell characteristics using flow cytometry (a technique that analyzes cell properties), and immune system features.

    If the disease returns, additional tests will be performed to detect any resistance mutations, which are genetic changes that may allow cancer cells to survive despite treatment.

    Specific tests will look for mutations in genes such as NRAS, KRAS, PTPN11, CBL, and NF1, as well as changes in proteins and cell surface markers.

  5. Step 5

    Contraception requirements during treatment

    If you are a female of childbearing potential, you must use an effective method of contraception during treatment with gilteritinib and for at least 6 months after your last dose.

    If you are using hormonal contraceptives, you should also use a barrier method of contraception.

    You must not be pregnant, breastfeeding, or considering becoming pregnant during the study or for at least 6 months after the last dose of gilteritinib.

    If you are a male who is sexually active with a female partner of childbearing potential, you must agree to use effective contraception during treatment and for at least 4 months after your last dose of gilteritinib.

  6. Step 6

    Completion of study treatment

    You will continue treatment and follow-up visits according to the study protocol.

    The duration of your participation will depend on your individual response to treatment and the study requirements.

    Final assessments will be performed to evaluate the overall effectiveness of the treatment and any long-term effects.

Who can join the trial?

14 criteria

  • You must provide signed and dated informed consent, which is a document showing you agree to participate in the study after understanding all the details, before any study procedures begin.
  • You must be an adult male or female patient between 18 and 75 years old at the time of signing the consent form.
  • You must have a new diagnosis of primary or de novo FLT3-ITD mutated AML, which means a newly identified type of acute myeloid leukaemia with a specific genetic change called FLT3-ITD, according to the 2017 WHO criteria.
  • You must have positive MRD, which stands for measurable residual disease, detected by a laboratory test called PCR-NGS for FLT3-ITD, after completing 2 cycles of intensive chemotherapy combined with an FLT3-inhibitor medication.
  • Your body weight must be more than 40 kilograms.
  • You must have an ECOG performance status of less than 2, which is a scale doctors use to measure how well you can carry out daily activities, with lower numbers meaning better ability to function.
  • Your estimated life expectancy must be more than 3 months.
  • If you are a female who can become pregnant, you must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test within seven days before starting treatment with Gilteritinib.
  • If you are a female who can become pregnant, you must agree to use an effective method of contraception, which is birth control, during treatment with Gilteritinib and for at least 6 months after stopping the medication.
  • If you are a female who can become pregnant and are using hormonal contraceptives, you should add a barrier method of contraception, which is a physical form of birth control such as condoms.
  • If you are a female who can become pregnant, you must not be pregnant, breastfeeding, or planning to become pregnant during the study or for at least 6 months after the last dose of Gilteritinib.
  • If you are a male who is sexually active with a female partner who can become pregnant, you must agree to use effective contraception during treatment and for at least 4 months after the last dose of Gilteritinib.
  • You must be willing and able to return to the clinical site for follow-up visits as required.
  • You must be willing and able to follow all study procedures and requirements as described in the study plan.

Who cannot join the trial?

3 criteria

  • The study information does not list specific reasons why a patient cannot participate in this clinical trial
  • You should discuss with your doctor whether this study is appropriate for you based on your individual medical situation
  • Your doctor will review your complete medical history and current health condition to determine if you can safely join this study
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Investigated drugs

Gilteritinib is a medication being tested in this study for patients with a specific type of blood cancer called acute myeloid leukemia. This medicine works by targeting and blocking certain proteins in cancer cells that help them grow and survive. In this trial, it is being used to try to eliminate any remaining cancer cells that may still be present in the body after initial treatment, even when they cannot be easily detected by standard tests.

What is already known about the treatment

Gilteritinib – Gilteritinib is a medication taken by mouth in tablet form that is used to treat acute myeloid leukemia, a type of blood cancer that affects white blood cells. It works by blocking specific proteins called FLT3 enzymes that help cancer cells grow and multiply, particularly in patients who have a mutation known as FLT3-ITD in their cancer cells. This medication belongs to a class of drugs called kinase inhibitors, which are targeted therapies that interfere with the signals cancer cells need to survive. Gilteritinib is approved for use in certain patients with acute myeloid leukemia and is being studied for its ability to eliminate remaining cancer cells that standard tests can detect after initial treatment.

Investigated diseases

Acute Myeloid Leukaemia – Acute myeloid leukaemia is a type of blood cancer that starts in the bone marrow, where blood cells are made. The disease occurs when immature white blood cells, called myeloblasts, grow out of control and crowd out healthy blood cells. These abnormal cells cannot function properly and prevent the bone marrow from producing normal red blood cells, white blood cells, and platelets. As the disease progresses, patients may experience fatigue, frequent infections, and easy bruising or bleeding due to low levels of healthy blood cells. The cancerous cells can accumulate in the blood and may spread to other parts of the body, including the liver, spleen, and lymph nodes. Without intervention, the condition worsens rapidly as abnormal cells continue to multiply and interfere with normal blood cell production.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-521875-30-00Protocol codeISR007028Estimated enrolment58 patientsSponsorHellenic Society Of Hematology

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