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Study Comparing Venetoclax and Azacitidine with Standard Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia Patients with NPM1 Mutations Eligible for Intensive Treatment

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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 patients who have a genetic change known as the NPM1 mutation. The study is comparing two treatment approaches for patients who are newly diagnosed with this condition and are eligible for intensive treatment. One group of patients will receive a combination of two medications: Venetoclax and Azacitidine. Venetoclax, also known by its code name ABT-199, is a medication that helps to kill cancer cells by blocking a protein that helps them survive. Azacitidine is a medication that works by stopping the growth of cancer cells. The other group will receive standard intensive chemotherapy, which is a common treatment for AML that uses strong drugs to kill cancer cells.

The purpose of this study is to compare the effectiveness of the combination of Venetoclax and Azacitidine with the standard intensive chemotherapy. Patients participating in the study will be randomly assigned to one of the two treatment groups. The study will monitor the patients over a period to see how well the treatments work in controlling the cancer and improving survival. The study will also look at the side effects of the treatments and how they affect the patients' quality of life.

Throughout the study, patients will receive regular check-ups and tests to monitor their health and the progress of the treatment. The study aims to provide valuable information that could help improve treatment options for patients with acute myeloid leukemia and the NPM1 mutation. Participation in the study is voluntary, and patients can choose to leave the study at any time. The study is expected to continue until 2028, with recruitment starting in 2024.

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 study, an initial assessment will be conducted to confirm eligibility. This includes a review of medical history and a physical examination.

    Blood tests will be performed to check for specific markers related to acute myeloid leukemia (AML) and to ensure adequate liver and kidney function.

  2. Step 2

    Treatment group assignment

    Participants will be randomly assigned to one of two treatment groups: the investigational group receiving venetoclax plus azacitidine, or the standard intensive chemotherapy group.

  3. Step 3

    Treatment initiation

    For those in the investigational group, treatment will begin with venetoclax taken orally as a film-coated tablet, alongside azacitidine administered subcutaneously (under the skin).

    Participants in the standard chemotherapy group will receive a combination of drugs including mitoxantrone, cytarabine, daunorubicin hydrochloride, and gemtuzumab ozogamicin, all administered intravenously (through a vein).

  4. Step 4

    Treatment cycles

    The investigational group will undergo up to three cycles of treatment, while the standard chemotherapy group will have up to two cycles.

    Each cycle involves a specific schedule of drug administration, followed by a period of rest to allow the body to recover.

  5. Step 5

    Monitoring and assessments

    Regular monitoring will occur throughout the trial to assess the effectiveness of the treatment and to check for any side effects.

    This includes blood tests, imaging studies, and other necessary evaluations to track progress and adjust treatment as needed.

  6. Step 6

    End of treatment evaluation

    At the end of the treatment period, a comprehensive evaluation will be conducted to determine the overall response to the therapy.

    This will include assessments of disease status and any changes in health-related quality of life.

  7. Step 7

    Follow-up

    Participants will continue to be monitored for a specified period after the treatment ends to observe long-term effects and overall survival.

    Follow-up visits will include routine check-ups and any additional tests deemed necessary by the study team.

Who can join the trial?

10 criteria

  • Must provide a signed informed consent, which means you agree to participate after understanding the study.
  • Must have been newly diagnosed with **CD33-positive acute myeloid leukemia (AML)** with an **NPM1 mutation** according to specific medical guidelines.
  • Must be between the ages of 18 and 70 years.
  • Must be fit for intensive chemotherapy, which means:
    • Have an **ECOG performance status** of 0 to 2, indicating you are fully active or have some restrictions but can care for yourself.
    • Have adequate liver function, meaning certain liver enzymes and bilirubin levels are not more than 2.5 times the normal limit, unless related to leukemia. If you have **Gilbert's Syndrome**, higher bilirubin levels may be acceptable after discussion with the study doctor.
    • Have adequate kidney function, shown by a serum creatinine level not more than 1.5 times the normal limit or a creatinine clearance rate of at least 50 mL/min, which measures how well your kidneys filter blood.
    • Must have a **white blood cell (WBC) count** of less than 25,000 per microliter. You may use a medication called hydroxyurea to meet this requirement.
    • Must be able to understand and willing to sign a written informed consent.
    • Male participants must agree to avoid unprotected sex and not donate sperm from the time of signing the consent until 7 months after the last dose of the study drug.
    • Women who can become pregnant must have a negative pregnancy test within 72 hours before the first dose of the study drug.

Who cannot join the trial?

9 criteria

  • Patients with any other type of cancer that is not acute myeloid leukemia cannot participate.
  • Patients who have received any other treatment for acute myeloid leukemia in the past cannot participate.
  • Patients with severe heart problems cannot participate.
  • Patients with severe liver problems cannot participate.
  • Patients with severe kidney problems cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have an active infection that requires treatment cannot participate.
  • Patients who have a known allergy to the study drugs cannot participate.
  • Patients who are unable to follow the study procedures cannot participate.
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Investigated drugs

Venetoclax is a medication used in this trial to help treat acute myeloid leukemia (AML). It works by targeting and blocking a specific protein in cancer cells that helps them survive. By blocking this protein, venetoclax can help kill the cancer cells and slow down the progression of the disease.

Azacitidine is another medication used in the trial for treating AML. It works by interfering with the growth of cancer cells. Azacitidine helps to stop the cancer cells from multiplying and can lead to their death, which may help in controlling the disease.

Standard intensive chemotherapy is a traditional treatment approach for AML. It involves using strong drugs to kill cancer cells. This type of therapy is called "intensive" because it uses high doses of chemotherapy drugs to try to eliminate as many cancer cells as possible. The goal is to achieve remission, where the signs and symptoms of cancer are reduced or disappear.

What is already known about the treatment

  • Venetoclax

    Venetoclax is taken orally in the form of a tablet and is currently being studied in combination with azacitidine for treating acute myeloid leukemia (AML) with specific genetic mutations. It is known for its ability to help kill cancer cells by targeting a protein called BCL-2, which helps cancer cells survive. Venetoclax is classified as a BCL-2 inhibitor and is being explored as a promising option for patients who are eligible for intensive treatment.

  • Azacitidine

    Azacitidine is administered through an injection under the skin or into a vein and is being evaluated in combination with venetoclax for the treatment of acute myeloid leukemia (AML). It works by incorporating into the DNA of cancer cells, disrupting their growth and leading to cell death. Azacitidine is classified as a hypomethylating agent and is used to improve outcomes in patients with specific genetic mutations in AML.

Investigated diseases

Acute myeloid leukemia – Acute myeloid leukemia is a type of cancer that affects the blood and bone marrow. 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 lead to symptoms such as fatigue, fever, 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 progression of acute myeloid leukemia can vary, with some patients experiencing a rapid decline in health. The disease requires careful monitoring to manage its progression and impact on the body.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515267-59-00Protocol codeTUD-VINC01-080Estimated enrolment146 patientsSponsorTechnische Universitaet Dresden

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).