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Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation

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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 two diseases: Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes with excess blasts-2 (MDS-EB2). These are types of blood cancers that affect the bone marrow and blood cells. The study is investigating the effectiveness of two treatments, gilteritinib and midostaurin, both of which are medications taken orally. Gilteritinib is also known by its code name, ASP2215. These treatments are being tested in combination with chemotherapy in patients who have a specific genetic mutation called the FLT3 mutation, which is known to affect the progression of these diseases.

The purpose of the study is to compare the effectiveness of gilteritinib and midostaurin when used alongside chemotherapy. Participants in the study will receive either gilteritinib or midostaurin, along with standard chemotherapy treatments. The study will include an initial phase of treatment to induce remission, followed by a consolidation phase to strengthen the remission, and then a maintenance phase lasting one year to help prevent the disease from returning. The study is designed to observe how well these treatments work in prolonging the time patients remain free from events like disease progression or relapse.

Throughout the study, participants will be monitored for their overall survival, which means the time they live from the start of the study, and for any side effects they may experience. The study will also look at how quickly patients recover their blood cell counts after each cycle of chemotherapy. The goal is to determine which treatment is more effective in managing these blood cancers in patients with the FLT3 mutation. The study is expected to continue until 2031, providing valuable information on the long-term benefits and safety of these treatments.

The research process

The trial runs in 5 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 such as age, health status, and genetic markers.

    Informed consent is required to participate in the study.

  2. Step 2

    Induction therapy

    Induction therapy involves the administration of either gilteritinib or midostaurin in combination with chemotherapy.

    Gilteritinib is provided as Xospata 40 mg film-coated tablets for oral use.

    Midostaurin is provided as Rydapt 25 mg soft capsules for oral use.

    The goal of this phase is to achieve complete remission by reducing the number of cancer cells.

  3. Step 3

    Consolidation therapy

    Following successful induction therapy, consolidation therapy is administered to eliminate any remaining cancer cells.

    The same medication used during induction therapy is continued in combination with chemotherapy.

  4. Step 4

    Maintenance therapy

    Maintenance therapy lasts for one year and involves continued administration of the assigned medication to prevent cancer recurrence.

    Regular monitoring is conducted to assess health status and response to treatment.

  5. Step 5

    End of study participation

    Upon completion of the maintenance therapy, participation in the study concludes.

    Final assessments are conducted to evaluate overall health and treatment outcomes.

Who can join the trial?

16 criteria

  • Must be at least 18 years old.
  • Must be able to take the study drug by mouth.
  • Must have a **WHO/ECOG performance status** of 2 or less. This is a scale that measures how well you can perform daily activities.
  • Must have **adequate liver function**, which means certain liver tests should be within acceptable limits unless affected by leukemia.
  • Must have **adequate kidney function**, meaning a creatinine clearance greater than 40 mL/min. This is a measure of how well your kidneys are working.
  • Must provide written informed consent, which means you agree to participate after understanding the study details.
  • Female participants must agree not to breastfeed during the study and for a specified time after the last dose of the study drug.
  • Female participants must not donate eggs during the study and for a specified time after the last dose of the study drug.
  • Must be able to give informed consent, meaning you understand and agree to the study's requirements.
  • Female participants must either be unable to have children or agree to use effective birth control methods during the study and for a specified time after the last dose of the study drug.
  • Male participants and their female partners must use effective birth control methods during the study and for a specified time after the last dose of the study drug.
  • Male participants must not donate sperm during the study and for a specified time after the last dose of the study drug.
  • Must agree not to join another study while participating in this one.
  • Must have newly diagnosed **Acute Myeloid Leukemia (AML)** or **MDS with excess of blasts-2 (EB2)**, with a specific **FLT3 gene mutation**. AML is a type of blood cancer, and MDS-EB2 is a related condition.
  • Must have a **FLT3 mutation**, which is a specific change in the FLT3 gene, confirmed by a test.
  • Must be considered eligible for **intensive chemotherapy**, which is a strong treatment for cancer.

Who cannot join the trial?

5 criteria

  • Patients who do not have a newly diagnosed condition of Acute Myeloid Leukemia (AML) with a FLT3 gene mutation. The FLT3 gene mutation is a specific change in the DNA that can affect how the leukemia behaves.
  • Patients who are not eligible for intensive chemotherapy. Intensive chemotherapy is a strong treatment that uses drugs to kill cancer cells.
  • Patients who have a different type of leukemia or blood disorder, such as MDS-EB2. MDS-EB2 is a type of blood disorder that can lead to leukemia.
  • Patients who are not within the specified age range for the study. The study is looking for participants within certain age groups.
  • Patients who are part of a vulnerable population that the study is not designed to include. Vulnerable populations may include groups like children or the elderly, who need special considerations.
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Investigated drugs

  • Gilteritinib

    is a medication used in this trial to treat patients with newly diagnosed Acute Myeloid Leukemia (AML) or Myelodysplastic syndromes with excess blasts-2 (MDS-EB2) who have a specific mutation in the FLT3 gene. It is being tested to see how well it works when combined with other treatments and used as a maintenance therapy for one year.

  • Midostaurin

    is another medication being tested in the trial. Like gilteritinib, it is used for patients with AML or MDS-EB2 who have the FLT3 gene mutation. The trial aims to compare the effectiveness of midostaurin when used with induction and consolidation therapy, followed by maintenance therapy, to see how it performs in improving patient outcomes.

What is already known about the treatment

  • Gilteritinib

    Gilteritinib is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in treating Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes with excess blasts-2 (MDS-EB2), particularly in patients with FLT3 mutations. The medication works by inhibiting the activity of the FLT3 protein, which is often overactive in these types of cancer, thereby slowing down or stopping the growth of cancer cells. Gilteritinib is classified as a tyrosine kinase inhibitor, a type of targeted cancer therapy.

  • Midostaurin

    Midostaurin is also taken orally in capsule form. It is being evaluated in clinical trials for its use in combination with other therapies for treating newly diagnosed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes with excess blasts-2 (MDS-EB2) in patients with FLT3 mutations. Midostaurin functions by blocking multiple kinases, including FLT3, which helps to prevent the proliferation of cancer cells. It belongs to the pharmacological class of multi-kinase inhibitors, used to target specific pathways involved in cancer cell growth.

Investigated diseases

  • Myelodysplastic Syndromes with Excess Blasts-2 (MDS-EB2)

    This is a type of blood disorder where the bone marrow produces an excessive number of immature blood cells, known as blasts. These immature cells do not function properly and can crowd out healthy blood cells, leading to symptoms like fatigue, infections, and bleeding. Over time, MDS-EB2 can progress to acute myeloid leukemia, a more aggressive form of blood cancer. The condition is characterized by having 10-19% of blasts in the bone marrow or blood. Patients may experience anemia, low white blood cell counts, and low platelet counts. The disease is considered a rare condition.

  • Acute Myeloid Leukemia (AML)

    This is a cancer of the blood and bone marrow characterized by the rapid growth of abnormal white blood cells. These cells accumulate in the bone marrow and interfere with the production of normal blood cells. AML can lead to symptoms such as fatigue, fever, frequent infections, and easy bruising or bleeding. The disease progresses quickly and requires prompt medical attention. It is classified based on the type of blood cell affected and the genetic mutations present. AML is also considered a rare disease.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-502478-18-00Protocol codeHO156Estimated enrolment777 patientsSponsorHemato-Oncologie voor Volwassenen Nederland (Hovon) Stichting

sourced from the EU Clinical Trials Register and site verification

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