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Study on the Safety and Effectiveness of Quizartinib for Children with Newly Diagnosed FLT3-ITD Positive AML and Normal NPM1

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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) in children and adolescents. Specifically, it looks at cases where the cancer cells have a particular genetic change known as FLT3-ITD and do not have another change called NPM1 mutation. The study is testing a treatment using a medication called Quizartinib, which is taken as an oral solution. Quizartinib is being tested both in combination with standard chemotherapy and on its own after high-dose therapy.

The purpose of the study is to evaluate the safety and effectiveness of Quizartinib in treating this specific type of AML. Participants will receive up to two courses of chemotherapy along with Quizartinib. The study will monitor how well the treatment works by checking if the cancer cells are reduced to very low levels in the bone marrow. This is done using a method called flow cytometry, which helps in measuring the number of cancer cells. The study will also assess the safety of Quizartinib by observing any side effects that occur during the treatment period.

Throughout the study, participants will be evaluated at different stages to see how their bodies respond to the treatment. This includes checking for any adverse effects and measuring the levels of cancer cells in the bone marrow. The study aims to find the most suitable dose of Quizartinib that can be safely given to children and adolescents with this type of AML. The overall goal is to improve treatment outcomes for young patients with this challenging form of leukemia.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be required to provide written informed consent or assent. This is a formal agreement to participate, which must be signed by you or your parent/legal guardian if you are a minor.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm eligibility. This includes checking your age, health status, and specific medical conditions. You must be between 1 month and 18 years old, with a specific type of leukemia known as FLT3-ITD positive and NPM1 wild-type AML.

  3. Step 3

    Medication administration

    You will receive a medication called Quizartinib, which is an oral solution. This means you will take it by mouth. The dosage and frequency will be determined by the study team based on your specific needs and the study protocol.

  4. Step 4

    Chemotherapy combination

    Quizartinib will be administered in combination with chemotherapy. This phase is designed to assess the safety and effectiveness of the treatment. The study will monitor your response to the treatment and any side effects.

  5. Step 5

    Monitoring and evaluation

    Throughout the study, regular evaluations will be conducted. These include checking your bone marrow to measure the level of leukemia cells and assess the treatment's effectiveness. This is done using a technique called flow cytometry.

  6. Step 6

    Safety assessment

    The study will also focus on safety by monitoring any side effects or adverse reactions to the treatment. This is crucial to determine the recommended dose for future patients.

  7. Step 7

    Completion of study

    Upon completion of the study, a final assessment will be conducted to evaluate the overall effectiveness and safety of the treatment. This will help in understanding the long-term benefits and risks associated with the medication.

Who can join the trial?

10 criteria

  • Must be enrolled in the CHIP-AML22/Master study.
  • Must have a specific type of leukemia called FLT3-ITD positive and NPM1 wild-type.
  • Age must be between 1 month and 18 years old at the time of initial diagnosis.
  • For those older than 16 years, a Karnofsky Performance status of more than 50% is required. For those 16 years or younger, a Lansky performance status score of more than 50% is needed. These scores measure the ability to perform daily activities.
  • Must have adequate organ function:
    • Renal (kidney) function: A calculated eGFR (a test of kidney function) of at least 50 mL/min/1.73 m².
    • Liver function: Total or direct bilirubin (a liver function test) less than 1.5 times the upper limit of normal for age, or less than 5 times if related to leukemia. AST and ALT (liver enzymes) less than 5 times the upper limit of normal, or less than 10 times if related to leukemia.
    • Must have a life expectancy of more than 6 weeks.
    • If applicable, a negative pregnancy test is required within 2 weeks before joining the quizartinib trial.
    • Must be able to swallow or take the medication quizartinib through a nasogastric (NG) tube, which is a tube that goes through the nose to the stomach.
    • Must provide written informed consent or assent for the quizartinib trial. For minors, consent must be given by parents or legal guardians according to local laws and regulations.

Who cannot join the trial?

6 criteria

  • Patients who do not have a newly diagnosed condition called **FLT3-ITD positive and NPM1 wild-type AML** cannot participate. This is a specific type of acute myeloid leukemia (AML) found in children.
  • Patients who have a different type of genetic mutation in their AML, other than **FLT3-ITD positive and NPM1 wild-type**, are not eligible.
  • Patients who are not within the specified age range for the study cannot participate. The study is for children.
  • Patients who are not able to safely tolerate the study medication, **quizartinib**, based on their health condition, are excluded.
  • Patients who have any other medical conditions that might interfere with the study treatment or its evaluation are not eligible.
  • Patients who are part of a vulnerable population that cannot safely participate in the study are excluded.
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Investigated drugs

Quizartinib is a medication being studied for its potential to help treat a type of blood cancer called acute myeloid leukemia (AML) in children. This medication is being tested to see if it can work well with chemotherapy and also on its own after high-dose therapy. The goal is to see if it can help reduce the number of cancer cells in the body, especially in children who have a specific genetic marker called FLT3-ITD and do not have another marker called NPM1 mutation. Researchers are also looking at how safe quizartinib is for children and what the best dose might be to use in future treatments.

What is already known about the treatment

Quizartinib – Quizartinib is administered orally and is currently being studied in clinical trials for its effectiveness and safety in treating a specific type of acute myeloid leukemia (AML) in children. This medication is particularly focused on patients with newly diagnosed pediatric AML that is positive for the FLT3-ITD mutation and lacks the NPM1 mutation. Quizartinib works by targeting and inhibiting the FLT3 protein, which is involved in the growth and survival of cancer cells, thereby helping to stop or slow down the progression of the disease. It is classified as a tyrosine kinase inhibitor, a type of drug that blocks specific enzymes involved in cancer cell growth.

Investigated diseases

Acute Myeloid Leukemia (AML) with FLT3-ITD Mutation and NPM1 Wild-Type – Acute Myeloid Leukemia (AML) is a type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells. In this specific form, there is a mutation in the FLT3 gene known as FLT3-ITD, which leads to uncontrolled cell division. The NPM1 gene remains in its normal, wild-type state, which can influence the disease's behavior and response to treatment. The disease progresses as these abnormal cells accumulate, interfering with the production of normal blood cells. This can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. Over time, the disease can advance rapidly, requiring prompt medical attention.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-505000-27-01Protocol codeMH22CAQEstimated enrolment68 patientsSponsorPrincess Maxima Center For Pediatric Oncology

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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).