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Study of Venetoclax and Azacitidine for Patients with Untreated Acute Myeloid Leukemia Who Cannot Have Standard Induction Therapy

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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). The study is investigating the effectiveness of a treatment that combines two medications: Venetoclax and Azacitidine. Venetoclax, also known by its code name ABT-199, is taken as a film-coated tablet, while Azacitidine is given as a powder for injection. The purpose of the study is to see if this combination can improve the overall survival and remission rates in patients who have not received treatment before and are not eligible for standard intensive chemotherapy.

Participants in the study will be randomly assigned to receive either the combination of Venetoclax and Azacitidine or Azacitidine with a placebo. A placebo is a substance with no active medication, used to compare the effects of the actual treatment. The study will be conducted over a period of time, with regular check-ups and assessments to monitor the participants' health and response to the treatment. The goal is to determine if the combination therapy can lead to better outcomes compared to Azacitidine alone.

Throughout the study, participants will be closely monitored by healthcare professionals to ensure their safety and well-being. The study aims to provide valuable information that could lead to improved treatment options for patients with Acute Myeloid Leukemia who are unable to undergo standard induction therapy due to age or other health conditions. This research could potentially offer new hope for those affected by this challenging disease.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial treatment phase

    The study begins with the administration of venetoclax and azacitidine. Venetoclax is taken orally in the form of a film-coated tablet. Azacitidine is administered either intravenously (IV) or subcutaneously (SC).

    The dosage and frequency of administration are determined by the study protocol and the healthcare provider overseeing the trial.

  2. Step 2

    Treatment cycles

    The treatment is organized into cycles. Each cycle typically lasts 28 days, during which the medications are administered according to the schedule provided by the healthcare team.

    The goal is to evaluate the effectiveness of the combination of venetoclax and azacitidine in improving overall survival and achieving complete remission or remission with incomplete marrow recovery.

  3. Step 3

    Monitoring and assessments

    Throughout the trial, regular monitoring and assessments are conducted to track progress and any side effects. This includes blood tests, physical examinations, and other necessary evaluations.

    Patient-reported outcomes, such as fatigue and quality of life, are also measured using specific questionnaires.

  4. Step 4

    Completion of treatment

    Upon completion of the treatment cycles, a final assessment is conducted to determine the overall response to the treatment.

    The primary endpoints of the study include the rate of complete remission and overall survival, while secondary endpoints focus on event-free survival and patient-reported outcomes.

Who can join the trial?

17 criteria

  • The patient must have a confirmed diagnosis of **Acute Myeloid Leukemia (AML)**, which is a type of blood cancer.
  • The patient must be unable to receive standard treatment due to age or other health conditions.
  • Female patients who can have children must have a negative pregnancy test before starting the study.
  • The patient must willingly sign a consent form approved by an ethics committee before any study procedures begin.
  • The patient must be at least 18 years old.
  • The patient must have a life expectancy of at least 12 weeks.
  • The patient must be considered unsuitable for intensive treatment if they are 75 years or older, or if they are 18 to 74 years old with certain health issues, such as:
    • A performance status score of 2 or 3, which measures the ability to perform daily activities.
    • A history of heart problems requiring treatment or certain heart function measurements.
    • Lung function measurements below a certain level.
    • Kidney function measurements between 30 and 45 mL/min.
    • Liver function measurements within a specific range.
    • Any other health condition that makes intensive treatment unsuitable, as judged by a doctor.
    • The patient must have a performance status score of 0 to 2 if they are 75 years or older, or 0 to 3 if they are 18 to 74 years old.
    • The patient must have adequate kidney function, shown by a creatinine clearance of at least 30 mL/min.
    • The patient must have adequate liver function, shown by specific liver enzyme and bilirubin levels, unless these are affected by leukemia.
    • Female patients must be either postmenopausal, surgically sterile, or using a specified method of birth control during the study and for 90 days after the last dose of the study drug.
    • Male patients who are sexually active must agree to use specified contraception methods during the study and for 90 days after the last dose of the study drug. They must also agree not to donate sperm during this time.

Who cannot join the trial?

7 criteria

  • Patients who have previously received treatment for Acute Myeloid Leukemia (AML) cannot participate. This means if you have already been treated for AML, you are not eligible.
  • Patients who have other serious health conditions that might interfere with the study or make it unsafe for them to participate are excluded. This includes conditions that could affect the study results or the patient's safety.
  • Patients who are unable to follow the study procedures or attend the required visits are not eligible. This means if you cannot commit to the study schedule, you cannot participate.
  • Patients who are pregnant or breastfeeding are not allowed to participate. This is to ensure the safety of both the mother and the baby.
  • Patients who are participating in another clinical trial at the same time are excluded. This is to avoid any interference between different study treatments.
  • Patients with a history of certain types of cancer, other than AML, may be excluded. This is to ensure that the study focuses on AML specifically.
  • Patients who have had a recent infection or illness that could affect their participation in the study are not eligible. This is to ensure the safety and accuracy of the study results.
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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, which can help to kill these cells and slow down the progression of the disease. In this study, venetoclax is combined with another medication to see if it can improve the survival and remission rates of patients who have not received previous treatment for AML.

  • Azacitidine

    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. Azacitidine is used in combination with venetoclax to see if this combination can be more effective in treating patients with AML who are not eligible for standard induction therapy.

What is already known about the treatment

  • Venetoclax

    Venetoclax is administered orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness in treating Acute Myeloid Leukemia (AML), particularly in patients who are not eligible for standard induction therapy. The main therapeutic indication for venetoclax is to improve overall survival and remission rates in AML patients. At the molecular level, venetoclax works by inhibiting a protein called BCL-2, which helps cancer cells survive; by blocking this protein, venetoclax promotes the death of these cancer cells. It is classified as a BCL-2 inhibitor in pharmacology.

  • Azacitidine

    Azacitidine is administered via injection, either subcutaneously or intravenously. It is an established medication used in the treatment of Acute Myeloid Leukemia (AML) and is being evaluated in combination with venetoclax to enhance treatment outcomes. The primary therapeutic use of azacitidine is to improve survival and remission rates in AML patients. Azacitidine works by incorporating into the DNA and RNA of cancer cells, disrupting their growth and leading to cell death. It is classified as a hypomethylating agent in pharmacology.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia (AML) 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. This disease progresses quickly and can lead to symptoms such as fatigue, fever, frequent infections, and easy bruising or bleeding. As the abnormal cells multiply, they can spread to other parts of the body, including the liver, spleen, and central nervous system. AML is more common in adults and can vary in its specific genetic and molecular characteristics. The progression of the disease can be aggressive, requiring prompt medical attention.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513631-25-00Protocol codeM15-656Estimated enrolment350 patientsSponsorAbbVie Deutschland GmbH & Co. KG

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