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Study on Midostaurin and Decitabine for Adults with Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Who Are Unfit for Intensive Treatment

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 two blood-related diseases: Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS). These are conditions where the bone marrow does not produce enough healthy blood cells. The study is testing a treatment that combines two medications: Midostaurin and Decitabine. Midostaurin is taken as a soft capsule, while Decitabine is given as an infusion, which means it is delivered directly into the bloodstream through a vein.

The purpose of the study is to see how well the combination of Midostaurin and Decitabine works in treating patients who are not fit for standard chemotherapy. The study will compare the effects of adding Midostaurin to a 10-day treatment with Decitabine. Participants will receive this treatment over three cycles, and the study will look at how many patients achieve a complete response, which means no signs of the disease are found after treatment.

During the study, researchers will also monitor the safety and tolerability of the treatment, which means they will check for any side effects and how severe they are. The study will also look at how long patients live without the disease getting worse and their overall survival. Additionally, the study will explore if certain gene mutations can predict how well patients respond to the treatment. The study is expected to continue until December 2026.

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

    Initial assessment

    Upon joining the clinical trial, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and performing necessary tests to ensure the patient meets the criteria for participation.

  2. Step 2

    Treatment cycle 1

    The first treatment cycle begins with the administration of decitabine. This medication is given as an infusion into a vein, known as intravenous use, over a period of 10 days.

    Following the decitabine treatment, midostaurin is taken orally. The dosage is 25 mg in the form of soft capsules, taken twice daily.

  3. Step 3

    Monitoring and evaluation

    Throughout the treatment cycle, regular monitoring is conducted to assess the patient's response to the medications. This includes blood tests and other evaluations to track progress and identify any side effects.

  4. Step 4

    Treatment cycle 2

    The second treatment cycle follows the same procedure as the first. Decitabine is administered intravenously for 10 days, followed by oral midostaurin at a dosage of 25 mg twice daily.

  5. Step 5

    Further monitoring

    After the second cycle, additional monitoring is performed to evaluate the effectiveness of the treatment and to check for any adverse effects.

  6. Step 6

    Treatment cycle 3

    The third treatment cycle is conducted in the same manner as the previous cycles, with decitabine given intravenously for 10 days and midostaurin taken orally at 25 mg twice daily.

  7. Step 7

    Final evaluation

    At the end of the third cycle, a comprehensive evaluation is performed to determine the overall response to the treatment. This includes assessing the cumulative complete response rate and any remaining symptoms.

Who can join the trial?

11 criteria

  • Must have a diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) with excess blasts, according to the WHO 2016 classification. This does not include acute promyelocytic leukemia.
  • Must be 18 years or older.
  • Must not be eligible for standard chemotherapy. This means either having a high score (HCT-CI ≥ 3) indicating other health issues or choosing not to undergo standard chemotherapy for personal reasons.
  • Must have a white blood cell (WBC) count of ≤ 30 x 109/L. If hydroxyurea was used to lower WBC, it should be stopped 2 days before starting decitabine treatment.
  • Must have adequate kidney and liver function, unless the disease affects these organs. This is shown by specific lab values:
    • Serum creatinine ≤ 221.7 µmol/L (≤ 2.5 mg/dL), unless related to AML.
    • Serum bilirubin ≤ 2.5 times the upper limit of normal, unless related to AML or due to Gilbert’s syndrome.
    • Alanine transaminase (ALT) ≤ 2.5 times the upper limit of normal, unless related to AML.
    • Must have a WHO performance status of 0, 1, or 2, which indicates the level of daily activity a person can perform.
    • Must be willing and able to use adequate contraception during the study and for 5 months after the last treatment.
    • Must provide written informed consent, meaning they understand the study and agree to participate.
    • Must be capable of giving informed consent, meaning they can understand the information and make decisions about their participation.

Who cannot join the trial?

9 criteria

  • Patients with other types of cancer that are not acute myeloid leukemia or myelodysplastic syndrome cannot participate.
  • Patients who have had a heart attack or severe heart problems in the past 6 months are not eligible.
  • Patients with uncontrolled high blood pressure cannot join the study.
  • Patients who are pregnant or breastfeeding are not allowed to participate.
  • Patients with severe liver or kidney disease cannot take part in the trial.
  • Patients who have an active infection that requires treatment with antibiotics are excluded.
  • Patients who have received another investigational drug within the last 30 days cannot participate.
  • Patients with a known allergy to the study drug or similar drugs are not eligible.
  • Patients who are unable to comply with the study procedures and follow-up visits are excluded.
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Investigated drugs

  • Midostaurin

    is a medication used in this clinical trial to help treat certain types of blood cancers, such as acute myeloid leukemia (AML) and high-risk myelodysplasia (MDS). It works by blocking specific proteins that cancer cells need to grow, which may help slow down or stop the cancer from spreading. In this study, midostaurin is being tested to see if it can improve the effectiveness of another treatment when used together.

  • Decitabine

    is another medication used in this trial. It is a type of chemotherapy that helps to stop the growth of cancer cells by interfering with their DNA. Decitabine is used to treat blood cancers like AML and MDS, and in this study, it is given over a period of 10 days. The trial is investigating whether adding midostaurin to decitabine can improve treatment outcomes for patients who are not fit for more intensive therapies.

What is already known about the treatment

Midostaurin – Midostaurin is administered orally in the form of capsules and is currently being studied in combination with decitabine for its potential benefits in treating acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). It is being evaluated in clinical trials to determine its effectiveness and safety when added to a 10-day decitabine regimen. The main therapeutic use of midostaurin is to help manage certain types of blood cancers by inhibiting specific proteins that promote cancer cell growth. It belongs to a class of drugs known as kinase inhibitors, which work by blocking the action of enzymes involved in the growth and spread of cancer cells.

Investigated diseases

  • Acute myeloid leukemia

    Acute myeloid leukemia is a type of cancer that starts in the blood-forming cells of the bone marrow. It progresses rapidly, leading to the accumulation of immature white blood cells called myeloblasts. These cells crowd out normal blood cells, causing symptoms like fatigue, frequent infections, and easy bruising or bleeding. As the disease advances, it can spread to other parts of the body, including the lymph nodes, liver, and spleen. The rapid progression requires prompt medical attention to manage symptoms and complications.

  • Myelodysplastic syndrome

    Myelodysplastic syndrome is a group of disorders caused by poorly formed or dysfunctional blood cells. It begins in the bone marrow, where blood cells are produced, leading to a shortage of healthy blood cells. This can result in symptoms such as fatigue, shortness of breath, and increased risk of bleeding and infections. Over time, the condition can worsen, with a potential to transform into acute myeloid leukemia. The progression varies among individuals, with some experiencing a slow course while others may have a more rapid decline in blood cell production.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-503829-18-00Protocol codeHO155Estimated enrolment140 patientsSponsorHemato-Oncologie voor Volwassenen Nederland (Hovon) Stichting

sourced from the EU Clinical Trials Register and site verification

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