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Study on Venetoclax, Azacitidine, and Donor Lymphocyte Infusion for Patients with Myelodysplastic Syndromes or Acute Myeloid Leukemia Relapse After Transplantation

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 the treatment of two blood-related diseases: Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML). These conditions affect the bone marrow, where blood cells are produced. The study involves a combination of treatments using the medications Venetoclax and Azacitidine, along with a procedure called donor lymphocyte infusion, which involves giving a patient white blood cells from a donor. The purpose of the study is to assess the safety and effectiveness of this treatment combination in patients who have experienced a relapse of MDS or AML after undergoing a stem cell transplant.

Participants in the study will receive the treatment over a period of time, with regular monitoring to ensure their safety and to evaluate how well the treatment is working. The study is divided into two phases. In the first phase, the focus is on understanding the safety profile of the treatment and determining the appropriate dose for the second phase. The second phase will concentrate on assessing how effective the treatment is in managing the diseases.

Throughout the study, participants will be closely observed for any side effects or changes in their condition. The study aims to provide valuable information on the potential benefits and risks of using Venetoclax and Azacitidine in combination with donor lymphocyte infusion for treating patients with relapsed MDS or AML after a stem cell transplant.

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

    Initiation of treatment

    Upon joining the study, the patient begins treatment with venetoclax and azacitidine. Venetoclax is administered orally in the form of film-coated tablets.

    The dosage and frequency of venetoclax will be determined based on the phase of the study and the patient's response to the treatment.

  2. Step 2

    Administration of azacitidine

    Azacitidine is given as a subcutaneous injection. The specific dosage and schedule will be provided by the healthcare team.

    The treatment aims to assess the safety and effectiveness of the combination of venetoclax and azacitidine.

  3. Step 3

    Monitoring and assessments

    Throughout the study, regular blood samples and assessments are conducted to monitor the patient's response to the treatment.

    These assessments help determine the safety profile and effectiveness of the treatment combination.

  4. Step 4

    Adjustment of treatment

    Based on the results of the assessments, the healthcare team may adjust the dosage of venetoclax and azacitidine to optimize treatment outcomes.

    The goal is to find the most effective dose with the least side effects.

  5. Step 5

    Completion of study

    The study is expected to continue until May 2026, with the duration of participation varying for each patient.

    Upon completion, the patient's overall response to the treatment will be evaluated according to established criteria.

Who can join the trial?

13 criteria

  • The patient must have a documented relapse of **MDS** (Myelodysplastic Syndromes) or **AML** (Acute Myeloid Leukemia) after a type of stem cell transplant called **allo-SCT** (allogeneic stem cell transplantation), with white blood cell count less than 15,000 per cubic millimeter.
  • The patient must be able to swallow capsules.
  • The patient must be 18 years of age or older.
  • The patient must have an **ECOG performance status** of 2 or less. This is a scale used to assess how a disease affects a patient's daily living abilities, with lower numbers indicating better function.
  • The patient must have adequate organ function, as shown by specific laboratory tests:
    • Serum creatinine less than 2 mg/dL or a calculated creatinine clearance of at least 30 mL/min for those with higher creatinine levels. Creatinine is a waste product in the blood that comes from muscle activity, and these tests check kidney function.
    • Serum total bilirubin should be 2.5 times the upper limit of normal (ULN) or less, or direct bilirubin should be at the ULN for those with higher total bilirubin levels. Bilirubin is a substance made during the normal breakdown of red blood cells, and these tests check liver function.
    • AST (SGOT) and ALT (SGPT) should be 2.5 times the ULN or less. These are enzymes that help check liver health.
    • Alkaline Phosphatase should be 5 times the ULN or less. If it's more than 2.5 times the ULN, the liver fraction should be 2.5 times the ULN or less. This enzyme is also used to check liver function.
    • The patient should not be resistant to platelet transfusions. Platelets are blood cells that help with clotting, and transfusions are given to increase platelet counts.
    • Female patients who can have children must use at least one method of birth control specified by the study, starting from the first day of the study until at least 30 days after the last dose of venetoclax or 3 months after the last dose of azacitidine, whichever is later.
    • Male patients who are sexually active with female partners who can have children must agree to use contraception specified by the study from the first dose of the study drug(s) until at least 30 days after the last dose of venetoclax or 3 months after the last dose of azacitidine, whichever is later.
    • The patient must be available for regular blood tests, study-related assessments, and appropriate medical care at the institution conducting the study for the entire duration of the study.
    • The patient must be able to understand and willing to sign a consent form that explains the investigational nature of the study.

Who cannot join the trial?

9 criteria

  • Patients with a history of other types of cancer that are not related to the study.
  • Patients who have had a heart attack or severe heart problems recently.
  • Patients with uncontrolled infections that are not responding to treatment.
  • Patients who are pregnant or breastfeeding.
  • Patients who have severe liver or kidney problems.
  • Patients who are unable to follow the study procedures or instructions.
  • Patients who have received another experimental treatment within a certain time frame before the study.
  • Patients with a known allergy to the study medication or its ingredients.
  • Patients who have a condition that the study doctors believe would make it unsafe for them to participate.
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Investigated drugs

  • Venetoclax

    is a medication used in this study to help treat certain types of blood cancers. It works by targeting and blocking a specific protein that cancer cells need to survive, which may help to kill the cancer cells or stop them from growing.

  • Azacitidine

    is another medication used in the study. It is a type of chemotherapy that helps to slow or stop the growth of cancer cells. It can also help to improve the function of bone marrow, which is important for producing healthy blood cells.

  • Donor Lymphocyte Infusion (DLI)

    is a therapy that involves giving a patient lymphocytes, a type of white blood cell, from a donor. This can help to boost the patient's immune system and improve its ability to fight cancer cells.

What is already known about the treatment

  • Venetoclax

    This medication is taken orally and is currently being studied in combination with azacitidine and donor lymphocyte infusion for treating myelodysplastic syndromes or acute myeloid leukemia with less than 30% marrow blasts. It is in the clinical trial phase, focusing on its safety and effectiveness in patients who have relapsed after stem cell transplantation. Venetoclax is primarily used for its ability to inhibit 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, a type of targeted cancer therapy.

  • Azacitidine

    Administered via injection, azacitidine is being evaluated in combination with venetoclax and donor lymphocyte infusion for its potential to treat myelodysplastic syndromes or acute myeloid leukemia with less than 30% marrow blasts. This medication is in the clinical trial phase, aiming to determine its safety and efficacy in patients who have relapsed after stem cell transplantation. Azacitidine works by incorporating into DNA and RNA, leading to the reactivation of genes that suppress tumor growth. It is classified as a hypomethylating agent, which helps to restore normal function to bone marrow cells.

Investigated diseases

  • Myelodysplastic Syndromes

    These are a group of disorders caused by poorly formed or dysfunctional blood cells. They occur when something goes wrong in the bone marrow, where blood cells are produced. Over time, the bone marrow becomes less effective at producing healthy blood cells, leading to symptoms like fatigue, infections, and easy bruising. The condition can progress slowly or quickly, and in some cases, it may transform into acute myeloid leukemia. The progression varies greatly among individuals, with some experiencing mild symptoms for years, while others may have a more rapid decline in blood cell production.

  • Acute Myeloid Leukemia

    This is a type of cancer that starts in the blood-forming cells of the bone marrow and quickly moves into the blood. 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. Symptoms can include fatigue, fever, frequent infections, and easy bruising or bleeding. The disease progresses quickly and requires prompt medical attention. It can affect people of all ages but is more common in older adults.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-514877-23-00Protocol codeVENTOGRAFTEstimated enrolment55 patientsSponsorGroupe Francophone Des Myelodysplasies

sourced from the EU Clinical Trials Register and site verification

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