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Study of venetoclax and azacitidine combination treatment for acute myeloid leukemia patients with measurable residual disease after chemotherapy or before stem cell transplant

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 study focuses on patients with Acute Myeloid Leukemia (AML) who still have measurable traces of cancer cells after initial chemotherapy or before stem cell transplantation. The study tests a combination treatment using two medications: venetoclax (taken as tablets by mouth) and azacitidine (given through injection under the skin or into a vein). This remaining trace amount of cancer cells is called Measurable Residual Disease (MRD).

The purpose of this research is to determine if the combination of venetoclax and azacitidine can eliminate these remaining cancer cells in AML patients. The treatment will be given in courses lasting up to 24 months. During the treatment, patients will receive venetoclax tablets daily (up to 400 mg per day) and azacitidine injections (75 mg per square meter of body surface).

The study includes two groups of patients: those who have a favorable type of AML and are not scheduled for stem cell transplantation, and those with other types of AML who are planned to receive a stem cell transplant. Regular tests will be performed to check if the cancer cells are still present in the bone marrow and blood during and after the treatment.

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 cycle

    You will receive a combination of two medications: azacitidine and venetoclax

    Azacitidine will be administered either through an intravenous (IV) line or as an injection under the skin

    Venetoclax will be taken as film-coated tablets by mouth

    The treatment will be organized in cycles

  2. Step 2

    Monitoring period

    Your blood cells and bone marrow will be regularly checked to measure the presence of remaining cancer cells (measurable residual disease or MRD)

    Blood tests will be performed to monitor your liver and kidney function

    The medical team will assess your general health status during each visit

  3. Step 3

    Treatment continuation

    Based on your response, you may receive between 2 to 4 courses of treatment

    Each course will follow the same medication pattern as the initial cycle

    Regular monitoring of your condition will continue throughout the treatment period

  4. Step 4

    Follow-up period

    After completing the treatment cycles, you will continue to be monitored for any changes in your condition

    Regular checks will be performed to assess if the cancer remains under control

    The follow-up period will continue according to the study protocol

Who can join the trial?

10 criteria

  • Must be at least 18 years old
  • Must have acute myeloid leukemia (AML) with either: - Persistent measurable residual disease (remaining cancer cells) or - Return of residual disease after initial chemotherapy - Or detectable disease before stem cell transplantation
  • Must have previously received at least one cycle of chemotherapy with drugs called cytarabine and anthracycline
  • Must have no signs of cancer spread to the brain or spinal cord
  • Must have good overall physical function (ECOG score of 2 or less)
  • Must have adequate kidney function (creatinine clearance of 30 mL/min or higher)
  • Must have adequate liver function with: - Liver enzymes (AST and ALT) no more than 3 times the normal limit - Bilirubin no more than 1.5 times the normal limit
  • Must be willing and able to sign an informed consent form
  • For men: Must use contraception during the study and for 3 months after the last dose
  • For women who can become pregnant: Must use two forms of contraception or practice complete abstinence: - For 28 days before starting treatment - Throughout the entire treatment - For 6 months after the last dose

Who cannot join the trial?

15 criteria

  • Age below 18 years or above 70 years
  • Previous treatment with venetoclax (a type of cancer medication)
  • Current active infection that requires intravenous antibiotics
  • Severe heart problems, including uncontrolled heart failure or recent heart attack
  • Severe liver problems (liver function tests more than 3 times the normal limit)
  • Severe kidney problems (requiring dialysis)
  • Pregnancy or breastfeeding
  • Active secondary cancer (another type of cancer present at the same time)
  • Participation in another clinical trial within the last 30 days
  • Mental conditions that prevent understanding of the study requirements
  • Known allergic reactions to study medications
  • Uncontrolled high blood pressure
  • History of significant bleeding in the last 6 months
  • Active central nervous system involvement of leukemia
  • Inability to take oral medications
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Investigated drugs

Venetoclax is a medication used to treat acute myeloid leukemia (AML). It works by blocking a protein that helps cancer cells survive. This medication helps eliminate remaining cancer cells that may not be visible through regular tests but can be detected through special testing (known as measurable residual disease or MRD). Azacitidine is a chemotherapy medication that works by helping to correct the function of abnormal cells in bone marrow. It is used in combination with venetoclax to treat acute myeloid leukemia. This medication helps to stop cancer cells from growing and may help destroy them. The combination of these two medications is used before stem cell transplantation to help eliminate any remaining cancer cells and improve treatment outcomes for patients with acute myeloid leukemia.

What is already known about the treatment

  • Venetoclax

    An oral medication used in combination therapy for acute myeloid leukemia (AML), administered as tablets once daily. This targeted therapy works by blocking the BCL-2 protein, which helps cancer cells survive, leading to their programmed cell death. Venetoclax belongs to the class of selective BCL-2 inhibitors and is particularly effective in treating patients with measurable residual disease after initial chemotherapy. The medication has shown promising results in clinical trials when combined with azacitidine, especially for patients who need additional treatment before stem cell transplantation.

  • Azacitidine

    A chemotherapy medication administered via subcutaneous injection or intravenous infusion, used in the treatment of acute myeloid leukemia (AML). It works by interfering with DNA synthesis and helping to restore normal blood cell production in the bone marrow through DNA hypomethylation. Azacitidine belongs to the class of hypomethylating agents and has demonstrated effectiveness in treating AML, particularly when combined with venetoclax for patients with measurable residual disease. The medication helps improve blood cell counts and can reduce the need for blood transfusions in patients with leukemia.

Investigated diseases

Acute Myeloid Leukemia (AML) - A type of blood and bone marrow cancer that affects the production of blood cells. The disease develops when the bone marrow produces abnormal white blood cells, known as myeloid cells, which grow rapidly and interfere with normal blood cell production. These abnormal cells accumulate in the bone marrow and blood, reducing the body's ability to produce healthy blood cells. The condition can progress quickly, affecting the production of red blood cells and platelets. Measurable residual disease (MRD) refers to the presence of small numbers of leukemia cells that may remain after treatment.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-510648-29-00Protocol codeCETLAM-LMA-AC-202401Estimated enrolment29 patientsSponsorGrupo Cooperativo De Estudio Y Tratamiento De Las Leucemias Agudas Y Mielodisplasias

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