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Study of Gilteritinib and Chemotherapy for Children, Adolescents, and Young Adults with Relapsed or Refractory Acute Myeloid Leukemia with FLT3 Mutation

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), specifically in children, adolescents, and young adults who have a mutation in the FLT3 gene. The study is testing a medication called Gilteritinib, also known by its code name ASP2215, in combination with chemotherapy. The chemotherapy regimen includes drugs such as Filgrastim, Fludarabine Phosphate, and Cytarabine. These medications are used to treat patients whose cancer has returned or has not responded to previous treatments.

The purpose of the study is to find the best dose of Gilteritinib when used with chemotherapy and to see how well this combination works in achieving remission, which means the cancer is no longer detectable. The study is divided into two phases. In the first phase, the focus is on determining the safest and most effective dose of Gilteritinib. In the second phase, the study looks at how many patients achieve complete remission after receiving the treatment. Participants will receive Gilteritinib in combination with chemotherapy over several cycles, and their response to the treatment will be closely monitored.

Throughout the study, researchers will also assess the safety and tolerability of the treatment, which means they will check for any side effects and how well patients can handle the medication. The study aims to provide valuable information on the effectiveness of Gilteritinib combined with chemotherapy in treating young patients with this specific type of AML. The trial is expected to continue until 2031, allowing researchers to gather comprehensive data on the treatment's impact.

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, eligibility is confirmed based on specific criteria, including age and the presence of a specific gene mutation related to acute myeloid leukemia (AML).

  2. Step 2

    Phase 1: dose escalation

    The initial phase involves determining the safest and most effective dose of gilteritinib when combined with chemotherapy. This phase is focused on finding the right balance between effectiveness and safety.

    During this phase, gilteritinib is administered orally in the form of film-coated tablets. The exact dosage is adjusted based on individual tolerance and response.

  3. Step 3

    Chemotherapy combination

    The chemotherapy regimen includes several medications: filgrastim (administered subcutaneously), fludarabine phosphate (administered intravenously), and cytarabine (administered both intrathecally and intravenously).

    These medications are given in a specific sequence to enhance the effectiveness of the treatment.

  4. Step 4

    Phase 2: dose expansion

    In this phase, the focus is on evaluating the complete remission rates after two cycles of gilteritinib combined with chemotherapy.

    The goal is to assess how well the treatment works in achieving remission in patients who have not responded to initial treatments or have relapsed.

  5. Step 5

    Monitoring and assessments

    Throughout the trial, regular monitoring is conducted to assess the safety and effectiveness of the treatment. This includes measuring the concentration of gilteritinib in the blood and evaluating the body's response to the treatment.

    Safety assessments are performed to ensure that any side effects are managed appropriately.

  6. Step 6

    End of trial

    The trial is expected to conclude by July 2031. At the end of the trial, the overall results will be analyzed to determine the success of the treatment in achieving remission and improving patient outcomes.

Who can join the trial?

5 criteria

  • The patient must have a positive test for the FLT3 mutation in their bone marrow or blood. This is a specific change in the genes that can be found in certain types of leukemia.
  • The patient must be between 6 months and less than 21 years old at the time of joining the study.
  • 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 in the bone marrow.
  • For the first part of the study, the patient must have had a return of the disease after treatment or not responded to initial treatment, with no more than one attempt to achieve remission.
  • For the second part of the study, the patient must not have responded to initial treatment or have had the disease return after the first successful treatment.

Who cannot join the trial?

10 criteria

  • Patients who have a different type of leukemia that is not **Acute Myeloid Leukemia (AML)**. AML is a type of cancer that affects the blood and bone marrow.
  • Patients who do not have the specific genetic marker called **FLT3/ITD**. This is a change in the DNA that can affect how the leukemia behaves.
  • Patients who have not experienced a return of their leukemia after treatment, known as a **relapse**, or whose leukemia has not stopped responding to treatment, known as **refractory**.
  • Patients who are not within the age range of children, adolescents, and young adults.
  • Patients who are not able to safely receive the study medication due to other health conditions.
  • Patients who are pregnant or breastfeeding.
  • Patients who are participating in another clinical trial at the same time.
  • Patients who have had a recent infection or other serious illness that could interfere with the study.
  • Patients who have had a recent surgery or are planning to have surgery during the study period.
  • Patients who have a history of other types of cancer that might affect the study results.
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Investigated drugs

  • Gilteritinib (ASP2215)

    is a medication being studied for its potential to treat a type of blood cancer called acute myeloid leukemia (AML). It is specifically being tested in children, adolescents, and young adults who have a genetic mutation known as FLT3/ITD, which makes their cancer more difficult to treat. Gilteritinib works by targeting and blocking the activity of the FLT3 protein, which is involved in the growth and survival of cancer cells. The goal of the study is to find the safest and most effective dose of gilteritinib when used in combination with chemotherapy.

  • FLAG Chemotherapy

    is a combination of chemotherapy drugs used to treat acute myeloid leukemia (AML). The name FLAG comes from the initials of the drugs used in this regimen: fludarabine, cytarabine (also known as Ara-C), and granulocyte colony-stimulating factor (G-CSF). This combination is used to help destroy cancer cells and improve the chances of remission in patients with AML. In this study, FLAG is used alongside gilteritinib to see if the combination can improve treatment outcomes for patients with relapsed or refractory AML.

What is already known about the treatment

Gilteritinib – Gilteritinib is administered orally and is currently being studied in clinical trials for its effectiveness in treating certain types of acute myeloid leukemia (AML), specifically those that are positive for FMS-like Tyrosine Kinase 3 (FLT3) mutations. It is primarily used for patients with relapsed or refractory AML. The medication works by inhibiting the activity of the FLT3 enzyme, which is involved in the growth and survival of cancer cells. Gilteritinib is classified as a tyrosine kinase inhibitor, a type of targeted cancer therapy.

Investigated diseases

Acute Myeloid Leukemia (AML) – Acute Myeloid Leukemia is a type of cancer that starts in the blood-forming cells of the bone marrow and quickly moves into the blood. It is characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. The disease progresses quickly and can spread to other parts of the body, including the lymph nodes, liver, spleen, and central nervous system. Symptoms may include fatigue, fever, frequent infections, and easy bruising or bleeding. AML is often classified based on genetic mutations, such as the FLT3/ITD mutation, which can influence the disease's behavior and response to treatment.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-512469-15-00Protocol code2215-CL-0603Estimated enrolment97 patientsSponsorAstellas Pharma Global Development Inc.

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