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Study on Midostaurin and Chemotherapy for Children with Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia

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 who have a mutation known as FLT3. The study is testing a treatment that combines a medication called midostaurin (also known by its code name PKC412) with standard chemotherapy. Chemotherapy is a common cancer treatment that uses drugs to kill cancer cells. The study aims to understand how safe and effective this combination is, as well as how the body processes midostaurin.

Participants in the study will receive midostaurin twice a day along with chemotherapy drugs such as cytarabine, etoposide, idarubicin, daunorubicin, mitoxantrone, and fludarabine. These drugs are given through a vein, which is known as intravenous use. After the initial treatment phase, participants will continue to receive midostaurin alone for a period to see if it helps keep the cancer from coming back. The study will monitor the safety of the treatment by checking for any side effects and how well the participants tolerate the medication.

The purpose of this study is to evaluate the safety and tolerability of midostaurin when used in combination with chemotherapy and as a standalone treatment after the initial therapy. The study will also look at how well the treatment works in reducing the cancer and preventing it from returning. Participants will be closely monitored throughout the study to ensure their safety and to gather information on how the treatment affects their health. The study is expected to continue until the end of 2029.

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 participant is confirmed to have a diagnosis of untreated FLT3-mutated Acute Myeloid Leukemia (AML).

    The participant may have received up to 7 days of hydroxyurea or low-dose cytarabine therapy before the first chemotherapy dose, if necessary.

  2. Step 2

    Initial chemotherapy

    The participant begins the first local induction chemotherapy as part of Block 1. This occurs while waiting for the results of the FLT3 analysis.

    Intrathecal chemotherapy may be administered as part of an initial diagnostic lumbar puncture or according to local standard care.

  3. Step 3

    Midostaurin treatment

    Once the FLT3 mutation is confirmed, the participant receives midostaurin in combination with standard chemotherapy.

    The dosage of midostaurin is 30 mg/m² twice daily or 1 mg/kg twice daily for participants under 10 kg body weight.

  4. Step 4

    Post-consolidation therapy

    After the initial chemotherapy, the participant undergoes 12 cycles of midostaurin as a single agent post-consolidation therapy.

    This phase aims to evaluate the safety and tolerability of midostaurin in combination with chemotherapy.

  5. Step 5

    Monitoring and assessments

    Throughout the trial, the participant's response to treatment is monitored through various assessments, including blood tests and imaging.

    The study evaluates the frequency and severity of any side effects, as well as the participant's overall response to the treatment.

  6. Step 6

    Completion of the trial

    The trial is estimated to end by December 30, 2029.

    Upon completion, the participant's overall survival and disease-free survival are assessed.

Who can join the trial?

8 criteria

  • The patient must have a documented diagnosis of a type of blood cancer called **Acute Myeloid Leukemia (AML)** that has not been treated before. This does not include a specific type called acute promyelocytic leukemia. The patient may have received up to 7 days of certain medications like hydroxyurea or low-dose cytarabine before starting the main treatment, if needed. They can also receive chemotherapy injected into the spinal fluid as part of initial tests or as per local care standards.
  • The patient must have a specific genetic change called a **FLT3 mutation**. This mutation must be confirmed by a special laboratory test before starting the study medication.
  • The patient must be between 3 months and less than 18 years old and expected to live for more than 12 weeks.
  • The patient must have a certain level of physical activity and ability to perform daily activities, measured by a score of 60 or higher on a scale called **Lansky** for those aged 1 to 16 years, or **Karnofsky** for those 16 years and older.
  • The patient must have certain laboratory test results that show their organs are working well enough. These include:
    • **AST and ALT** levels (liver enzymes) should be no more than 3 times the normal limit.
    • **Serum total bilirubin** should be no more than 1.5 times the normal limit, unless the patient has a condition called **Gilbert’s syndrome**.
    • **Creatinine clearance** (a measure of kidney function) should be at least 30 mL/min.
    • The patient or their parent/legal guardian must provide written consent to participate in the study, following local laws and regulations, before any study-related procedures are done.

Who cannot join the trial?

6 criteria

  • Participants who have not been diagnosed with **FLT3-mutated Acute Myeloid Leukemia**. This is a specific type of blood cancer with a particular genetic change.
  • Participants who have already received treatment for their **FLT3-mutated Acute Myeloid Leukemia**.
  • Participants who are not within the age range specified for the study. The study is for children, so adults cannot participate.
  • Participants who are not able to safely receive the study medication or chemotherapy due to other health conditions.
  • Participants who are pregnant or breastfeeding, as the study medication may affect the baby.
  • Participants who are unable to follow the study procedures or attend the required visits.
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Investigated drugs

Midostaurin is a medication being studied for its safety and effectiveness in children with a specific type of leukemia called FLT3-mutated acute myeloid leukemia (AML). In this trial, midostaurin is given in combination with standard chemotherapy to see if it can improve treatment outcomes. After the initial treatment phase, midostaurin is also used alone as a follow-up therapy to help prevent the cancer from returning. The goal is to determine how well children tolerate this medication and how it affects their cancer.

What is already known about the treatment

Midostaurin – This medication is administered orally, typically in the form of capsules, and is taken twice daily. It is currently being studied in clinical trials for its effectiveness and safety in treating children with newly diagnosed FLT3-mutated Acute Myeloid Leukemia (AML). Midostaurin is primarily used in combination with standard chemotherapy and as a standalone treatment after initial therapy. It works by inhibiting certain enzymes, specifically targeting the FLT3 mutation, which plays a role in the growth and survival of cancer cells. Midostaurin 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 progresses to affect 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 can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding due to the lack of healthy blood cells. AML is classified into different subtypes based on genetic mutations, such as the FLT3 mutation, which can influence the disease's behavior and response to treatment. The progression of AML can vary, but it generally requires prompt medical attention to manage the rapid increase of abnormal cells.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IITrial ID2023-509834-20-00Protocol codeCPKC412A2218Estimated enrolment22 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

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