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Study of venetoclax and azacitidine treatment in adults with NPM1-mutated acute myeloid leukemia who experience molecular relapse or disease progression

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 that has a specific genetic change called NPM1 mutation. The study aims to test if combining two medications - venetoclax (taken as tablets by mouth) and azacitidine (given as an injection under the skin) - can help prevent the cancer from returning in patients who show early signs of disease comeback at the molecular level.

The treatment involves taking venetoclax tablets daily and receiving azacitidine injections according to a specific schedule. The combination therapy will be given to patients who have already received standard chemotherapy treatment and achieved initial remission but are showing early signs that the disease might be returning based on specialized laboratory tests.

During the study, patients will be monitored regularly to check how well the treatment is working and to watch for any side effects. The main goal is to see if this drug combination can stop the disease from progressing to a full relapse and allow patients to proceed to stem cell transplantation, which is a procedure that replaces damaged bone marrow with healthy cells.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial evaluation

    Your eligibility for the study will be confirmed through medical tests and evaluation of your medical history.

    The study focuses on patients with Acute Myeloid Leukemia who have a specific genetic mutation called NPM1.

    You must be at least 18 years old and have adequate kidney and liver function to participate.

  2. Step 2

    Treatment initiation

    The treatment combines two medications:

    Venetoclax - taken by mouth

    Azacitidine - given as an injection under the skin

  3. Step 3

    Regular monitoring - 3 months

    Your response to treatment will be evaluated

    Medical tests will check for signs of disease and treatment effectiveness

    Your blood and bone marrow will be tested to measure the level of leukemia cells

  4. Step 4

    6-month evaluation

    A comprehensive assessment will determine if the disease has returned

    Additional tests will measure the levels of leukemia cells in your body

    Your eligibility for stem cell transplant will be evaluated

  5. Step 5

    Stem cell transplant preparation

    If appropriate, preparation for stem cell transplant will begin

    Your treatment response will be evaluated before proceeding with transplant

  6. Step 6

    Long-term follow-up

    Your health will be monitored for up to 12 months

    Regular evaluations will check for disease progression

    Overall survival and treatment effectiveness will be assessed

Who can join the trial?

14 criteria

  • Must be 18 years of age or older
  • If female and able to have children, must have a negative pregnancy test during screening
  • If able to have children (both men and women), must agree to use effective contraception during the study and for 3 months after treatment ends
  • Must provide written informed consent to participate in the study
  • Must have been previously diagnosed with NPM1-mutated Acute Myeloid Leukemia, with or without additional genetic mutations (FLT3-TKD or FLT3-ITD)
  • Must have confirmed NPM1 type A, B, or D mutations during screening
  • Must be eligible for stem cell transplantation according to transplant center requirements
  • Must have completed at least two cycles of standard chemotherapy and achieved first complete remission
  • Must be in first complete remission with detectable minimal residual disease (very small amounts of cancer cells) in bone marrow
  • Must have a life expectancy of at least 12 weeks
  • Must have good physical function status (ECOG score less than 2, meaning able to carry out light work)
  • Must have adequate liver function:
    • Liver enzymes (AST and ALT) less than 3 times the normal limit
    • Bilirubin less than 1.5 times the normal limit
    • Must have adequate kidney function (creatinine clearance of 30 mL/min or higher)

Who cannot join the trial?

14 criteria

  • Age below 18 years or above 75 years
  • Patients without confirmed NPM1 mutation (a specific genetic change in blood cells)
  • Active or uncontrolled infections
  • Previous treatment with venetoclax or azacitidine (medications used in the study)
  • Pregnant or breastfeeding women
  • Severe kidney problems (creatinine clearance less than 30 mL/min)
  • Severe liver problems
  • History of other cancers in the past 2 years (except treated non-melanoma skin cancer or cervical cancer in situ)
  • Uncontrolled heart conditions
  • Mental conditions that prevent understanding of study requirements
  • Participation in other clinical trials within 30 days before this study
  • Known allergic reactions to study medications
  • Active central nervous system involvement of leukemia
  • HIV, hepatitis B, or active hepatitis C infection
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Investigated drugs

  • Venetoclax

    is a medication that works by blocking a protein called BCL-2, which helps cancer cells survive. It is used to treat blood cancers. In this trial, it is being studied as a treatment for acute myeloid leukemia (AML) to prevent the disease from coming back.

  • Azacitidine

    is a chemotherapy medication that works by helping bone marrow produce normal blood cells and by killing abnormal cells in the blood and bone marrow. It is commonly used to treat certain blood disorders and blood cancers. In this study, it is being combined with venetoclax to treat patients with AML who show early signs of disease return at the molecular level.

What is already known about the treatment

  • Venetoclax

    An oral medication used in the treatment of blood cancers, particularly acute myeloid leukemia with NPM1 mutation. It works by blocking the BCL-2 protein, which helps cancer cells survive, leading to their programmed cell death. This targeted therapy belongs to the class of drugs known as BCL-2 inhibitors and is typically administered in tablet form as part of combination therapy with other anti-cancer medications.

  • Azacitidine

    A chemotherapy medication administered either by subcutaneous injection or intravenous infusion, used in treating acute myeloid leukemia and other blood disorders. It functions by blocking DNA methylation, helping to restore normal function to genes that control cell growth and differentiation. This hypomethylating agent is classified as a nucleoside metabolic inhibitor and is often used in combination therapy for patients who cannot undergo intensive chemotherapy.

Investigated diseases

Acute Myeloid Leukemia with NPM1 mutation - A specific type of blood cancer that affects the bone marrow, characterized by a genetic mutation in the NPM1 gene. This condition causes the bone marrow to produce abnormal white blood cells that don't mature properly. The abnormal cells accumulate in the bone marrow and interfere with the production of normal blood cells. The NPM1 mutation affects a protein that normally helps control cell growth and division. This form of leukemia can develop quickly, with symptoms including fatigue, frequent infections, and easy bruising or bleeding.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-510432-36-00Protocol codeAML2521Estimated enrolment35 patientsSponsorFondazione Gimema Franco Mandelli Onlus

sourced from the EU Clinical Trials Register and site verification

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