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Study on Stopping Venetoclax and Azacitidine in Patients with Acute Myeloid Leukemia Who Are Responding to 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 acute myeloid leukemia (AML), a type of cancer that affects the blood and bone marrow. The study involves two medications: Azacitidine, which is given as a powder for suspension for injection, and Venetoclax, available as film-coated tablets. Venetoclax is also known by its code names ABT-199 and GDC-0199. The purpose of the study is to evaluate the effects of stopping these medications in patients who have responded well to treatment.

Participants in the study will have been receiving a combination of Azacitidine and Venetoclax for about a year. The study will observe what happens when these medications are gradually reduced and eventually stopped. Researchers will monitor the participants' health to see how long they remain free of the disease after stopping the treatment. This period is known as Disease-Free Survival. The study will also look at other factors, such as the duration of the response to treatment and overall survival.

Throughout the study, participants will be regularly checked to ensure their health and safety. The study aims to gather information that could help improve future treatment strategies for patients with acute myeloid leukemia. By understanding how patients respond to the discontinuation of these medications, researchers hope to find ways to maintain health and quality of life without continuous treatment.

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, the patient will begin the process of VEN-AZA de-escalation. This involves gradually reducing the use of two medications: azacitidine and venetoclax.

    The patient will have been receiving these medications as part of their initial treatment for acute myeloid leukemia (AML).

  2. Step 2

    Medication administration

    Azacitidine is administered as a suspension for injection under the skin (subcutaneous use).

    Venetoclax is taken as film-coated tablets by mouth (oral use).

    The specific dosage and frequency of these medications will be determined by the healthcare provider based on the patient's response to treatment.

  3. Step 3

    Monitoring and assessments

    Throughout the trial, the patient's health and response to the medication de-escalation will be closely monitored.

    Regular assessments will be conducted to measure disease-free survival, which is the time from the start of the de-escalation to any signs of disease returning or other health events.

  4. Step 4

    Follow-up and evaluation

    The patient will have follow-up visits to evaluate the effectiveness of the de-escalation strategy.

    These evaluations will include checking for any signs of relapse, monitoring overall health, and assessing quality of life using a standardized questionnaire.

  5. Step 5

    Completion of the trial

    The trial is expected to continue until November 2028, with each patient's participation duration varying based on individual response and health status.

    Upon completion, the patient's overall health and treatment outcomes will be reviewed to determine the success of the de-escalation strategy.

Who can join the trial?

11 criteria

  • Must be a female or male who is 18 years or older.
  • Must be affiliated with the French Social Security or have similar health insurance.
  • Must have signed informed consent, meaning you agree to participate after understanding the study.
  • Must have a diagnosis of previously untreated acute myeloid leukemia (AML) according to the 2022 classification.
  • Must have received VEN-AZA as the first treatment for AML.
  • Must have had 12 months of VEN-AZA therapy, with some flexibility in the exact duration and doses.
  • Must be in first composite complete response, which means a complete response or partial recovery of blood cells.
  • Must have no detectable minimal residual disease (MRD), meaning very low levels of cancer cells, as tested locally.
  • Must have an ECOG score of less than 3, which is a measure of your ability to perform daily activities.
  • Females of childbearing potential must have a negative pregnancy test and agree to use effective birth control for 6 months after stopping VEN-AZA. Male patients must agree to use effective birth control for 3 months after stopping VEN-AZA.
  • Must not have any psychological, familial, sociological, or geographical conditions that could interfere with following the study protocol and schedule.

Who cannot join the trial?

10 criteria

  • Patients with any other type of cancer besides acute myeloid leukemia cannot participate.
  • Patients who have had a bone marrow or stem cell transplant in the past cannot participate.
  • Patients with severe heart problems cannot participate.
  • Patients with uncontrolled infections cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are currently participating in another clinical trial cannot participate.
  • Patients with a history of allergic reactions to the study drugs cannot participate.
  • Patients with severe liver or kidney problems cannot participate.
  • Patients who have received certain treatments for leukemia in the past 2 weeks cannot participate.
  • Patients with any condition that the study doctors believe would make it unsafe for them to participate cannot participate.
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Investigated drugs

  • Venetoclax

    is a medication used in this clinical trial. It works by helping to kill cancer cells. It does this by blocking a protein that cancer cells need to survive. In this study, the researchers are looking at what happens when patients stop taking Venetoclax after their cancer has responded to treatment.

  • Azacitidine

    is another medication used in the trial. It helps to stop cancer cells from growing and dividing. Azacitidine is often used to treat certain types of blood cancers. In this study, the researchers are examining the effects of stopping Azacitidine in patients whose cancer has responded to treatment.

What is already known about the treatment

  • Venetoclax

    Venetoclax is taken orally in the form of a tablet and is currently being studied in clinical trials for its effectiveness in treating acute myeloid leukemia (AML). It is a medication that is already used in medicine, particularly for certain types of blood cancers. The main purpose of using Venetoclax is to help patients with AML by stopping the growth of cancer cells. It works at the molecular level by blocking a protein that helps cancer cells survive, leading to their death. Venetoclax is classified as a BCL-2 inhibitor, which is a type of drug that targets specific proteins in cancer cells.

  • Azacitidine

    Azacitidine is administered through an injection under the skin or into a vein and is being evaluated in clinical trials for its role in treating acute myeloid leukemia (AML). It is a well-established medication in the medical field, primarily used for certain blood disorders and cancers. Azacitidine helps treat AML by interfering with the growth of cancer cells and promoting their death. At the molecular level, it works by incorporating into the DNA of cancer cells, disrupting their function and growth. Azacitidine is classified as a hypomethylating agent, which means it helps to modify the DNA in a way that can stop cancer cells from multiplying.

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 of the disease requires prompt medical attention to manage symptoms and complications.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-514112-28-00Protocol codeIPC 2024-001Estimated enrolment50 patientsSponsorInstitut Paoli Calmettes

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