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Study on the Safety and Effectiveness of Quizartinib for Children and Young Adults with Acute Myeloid Leukemia (AML)

Verified siteInvestigationalNo 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), which affects children and young adults. The study is investigating a treatment using a medication called Quizartinib, also known by its code name AC220. Quizartinib is a type of drug known as a Class III receptor tyrosine kinase inhibitor, which is designed to target specific mutations in cancer cells. The trial aims to evaluate the safety and effectiveness of Quizartinib when used in combination with chemotherapy and as a standalone treatment.

The purpose of the study is to determine the best dose of Quizartinib for different age groups and to assess how well it works in achieving remission, which means the cancer is no longer detectable. The study will also look at the safety of the treatment and how the body processes the medication. Participants will receive Quizartinib along with chemotherapy for up to two cycles, with the option for additional treatment if needed. After this, Quizartinib may be given alone for up to twelve cycles to see if it helps keep the cancer from returning.

This trial is specifically for patients with a mutation called FLT3-ITD, which is found in some cases of AML. The study will monitor how well the treatment works in reducing cancer cells and how long the remission lasts. It will also track any side effects experienced by participants. The goal is to find a safe and effective treatment option for young 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

    Joining the study

    Upon joining the study, the patient is confirmed to have a diagnosis of acute myeloid leukemia (AML) with a specific genetic mutation known as FLT3-ITD.

    The patient is between 1 month and 21 years old and has met all the necessary health criteria to participate.

  2. Step 2

    Re-induction cycle 1

    The patient begins the first cycle of treatment, known as Re-Induction Cycle 1.

    During this cycle, the patient receives a combination of chemotherapy drugs and quizartinib, an oral solution taken by mouth.

    The goal is to achieve a state called complete remission (CR) or CR with incomplete recovery (CRi).

  3. Step 3

    Re-induction cycle 2

    If necessary, the patient may undergo a second cycle of re-induction treatment.

    The same combination of chemotherapy and quizartinib is administered to further reduce cancer cells.

  4. Step 4

    Optional consolidation therapy

    Following the re-induction cycles, the patient may receive additional chemotherapy, known as consolidation therapy, to help maintain remission.

  5. Step 5

    Single-agent continuation therapy

    The patient transitions to a phase where only quizartinib is administered.

    This phase can last up to 12 cycles, aiming to sustain remission and prevent relapse.

  6. Step 6

    Monitoring and follow-up

    Throughout the trial, the patient's health and response to treatment are closely monitored.

    Regular assessments are conducted to evaluate the effectiveness and safety of the treatment.

Who can join the trial?

13 criteria

  • The patient must have a diagnosis of **Acute Myeloid Leukemia (AML)**, which is a type of blood cancer, with at least 5% of abnormal cells (blasts) in the bone marrow.
  • Male patients must be surgically sterile or agree to use highly effective birth control during treatment and for 6 months after the last dose of the study drugs.
  • The patient or their legal representative must understand the study's experimental nature, potential risks, and benefits, and sign a written consent form.
  • Female patients must not donate or retrieve eggs for their own use from the time of screening and throughout the treatment period, and for at least 6 months after the last dose.
  • Male patients must not freeze or donate sperm starting at screening and throughout the treatment period, and for at least 6 months after the last dose.
  • The patient must be in their first relapse or not responding to the first round of high-dose chemotherapy, with no more than one attempt at remission induction. Previous **Hematopoietic Stem Cell Transplant (HSCT)** is allowed.
  • The patient must have a specific genetic mutation called **FLT3-ITD** in their bone marrow or blood.
  • The patient must be between 1 month and 21 years old at the time of signing the consent form.
  • The patient must have a performance status score that meets the study's requirements, indicating they are well enough to participate.
  • The patient must have fully recovered from any significant side effects of previous cancer treatments before starting the new treatment cycle.
  • The patient must have adequate kidney, liver, and heart function as defined by the study.
  • If the patient can have children, they must be permanently sterile or agree to use highly effective birth control during the study and for 6 months after the last dose of the study drugs.
  • If the patient is a female who can become pregnant, she must test negative for pregnancy and agree not to breastfeed during the study.

Who cannot join the trial?

7 criteria

  • Patients who have a different type of cancer other than Acute Myeloid Leukemia cannot participate. Acute Myeloid Leukemia is a type of blood cancer.
  • Patients who are not within the specified age range of 1 month to 21 years old are excluded.
  • Patients who are not able to safely receive the study medication or chemotherapy due to other health conditions are excluded.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may be excluded.
  • Patients who have certain infections or other serious health issues that could interfere with the study are excluded.
  • Patients who are unable to follow the study procedures or attend required visits are excluded.
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Investigated drugs

Quizartinib is a medication being studied for its potential use in treating pediatric patients with a specific type of leukemia known as acute myeloid leukemia (AML) that has returned or is resistant to treatment. This medication is being tested in combination with chemotherapy to see if it can help improve the effectiveness of the treatment. Quizartinib is also being evaluated as a standalone therapy to see if it can help maintain remission in patients after they have completed chemotherapy. The study aims to find the best dose of quizartinib for different age groups and to understand how the body processes the medication.

What is already known about the treatment

Quizartinib – Quizartinib is administered orally and is currently being studied in clinical trials for its use in treating Acute Myeloid Leukemia (AML), particularly in pediatric patients with FLT3-ITD mutations. It is in the investigational stage, with ongoing research to determine its safety and effectiveness when combined with chemotherapy and as a standalone treatment. The main therapeutic indication is for relapsed or refractory AML in young patients. Quizartinib works by inhibiting the FLT3 receptor, which is involved in the growth and survival of cancer cells. It is classified as a tyrosine kinase inhibitor, targeting specific proteins that contribute to cancer cell proliferation.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia (AML) is a type of cancer that starts in the bone marrow, where blood cells are made, and quickly moves into the blood. It affects a group of white blood cells called myeloid cells, which normally develop into various types of mature blood cells. In AML, these cells do not mature properly and multiply rapidly, leading to a buildup of abnormal cells that can crowd out normal blood cells. This can result in symptoms such as fatigue, frequent infections, and easy bruising or bleeding. The disease can progress quickly, requiring prompt medical attention. AML is more common in adults but can also occur in children.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-510009-16-00Protocol codeAC220-A-U202Estimated enrolment20 patientsSponsorDaiichi Sankyo Inc.

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