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Study on Venetoclax and Azacitidine for Patients Newly Diagnosed with Higher-Risk Myelodysplastic Syndrome

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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 condition known as Higher-Risk Myelodysplastic Syndrome (MDS). MDS is a group of disorders caused by poorly formed or dysfunctional blood cells. The study is evaluating the effectiveness and safety of a treatment that combines two medications: Venetoclax and Azacitidine. Venetoclax is a medication that works by blocking a protein that helps cancer cells survive, while Azacitidine is used to help the bone marrow produce normal blood cells and reduce the need for blood transfusions.

The purpose of this study is to determine if adding Venetoclax to the standard treatment with Azacitidine can improve the overall survival of patients who have been newly diagnosed with higher-risk MDS. Participants in the study will be randomly assigned to receive either the combination of Venetoclax and Azacitidine or a placebo with Azacitidine. 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, and participants will receive the treatment in cycles, with regular monitoring by healthcare professionals to assess their response to the treatment.

Throughout the study, various aspects of the participants' health will be monitored, including their overall response to the treatment, any improvements in blood cell counts, and their need for blood transfusions. The study will also look at how the treatment affects participants' physical functioning and fatigue levels. The main goal is to see if the combination of Venetoclax and Azacitidine can help patients live longer and improve their quality of life compared to the standard treatment alone.

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 assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a diagnosis of Higher-Risk Myelodysplastic Syndrome (MDS) with less than 20% bone marrow blasts.

    Eligibility criteria include being 18 years or older, having no prior therapy for MDS, and meeting specific disease activity criteria.

  2. Step 2

    Randomization

    Participants are randomly assigned to one of two groups: one receiving venetoclax and azacitidine, and the other receiving a placebo with azacitidine.

    This process is double-blind, meaning neither the participants nor the researchers know which group the participants are in.

  3. Step 3

    Treatment phase

    Participants in the venetoclax group take the medication orally in the form of film-coated tablets.

    Azacitidine is administered as a suspension for injection.

    The treatment aims to improve overall survival and is monitored for safety and efficacy.

  4. Step 4

    Monitoring and follow-up

    Participants undergo regular monitoring to assess the treatment's impact on their condition.

    Primary and secondary endpoints include overall survival, response rates, and quality of life measures.

    The study is expected to continue until October 31, 2029, with ongoing assessments throughout the trial duration.

Who can join the trial?

8 criteria

  • Participants must be **18 years or older**.
  • Participants must have a diagnosis of **Myelodysplastic Syndrome (MDS)** according to the 2016 World Health Organization (WHO) classification. MDS is a group of disorders caused by poorly formed or dysfunctional blood cells.
  • Participants must have less than **20% bone marrow blasts**. Bone marrow blasts are immature blood cells found in the bone marrow.
  • Participants must not have received any previous treatment for MDS with **hypomethylating agents**, chemotherapy, or **allogeneic stem cell transplantation**. Hypomethylating agents are drugs used to treat certain blood disorders, and allogeneic stem cell transplantation is a procedure where a person receives blood-forming stem cells from a donor.
  • Participants must have an **Overall Revised International Prognostic Scoring System (IPSS-R) score** greater than 3, which indicates intermediate, high, or very high disease activity. The IPSS-R is a tool used to assess the severity of MDS.
  • Participants must have an **Eastern Cooperative Oncology Group (ECOG) performance status** of 2 or less. The ECOG performance status is a scale used to assess how a disease affects a patient's daily living abilities, with 0 being fully active and 5 being deceased.
  • Participants must be eligible for a **hematopoietic stem cell transplant (HSCT)** but should not have a pre-arranged HSCT at the start of the study. HSCT is a procedure to replace damaged or destroyed bone marrow with healthy bone marrow stem cells.
  • If participants are not eligible for HSCT, they should not have plans for HSCT at the start of the study.

Who cannot join the trial?

6 criteria

  • Patients who have already received treatment for their **Higher-Risk Myelodysplastic Syndrome** cannot participate. This means if you have been treated for this condition before, 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 worsen with the study treatment.
  • Patients who are pregnant or breastfeeding are not allowed to join the study. This is to ensure the safety of both the mother and the baby.
  • 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 have participated in another clinical trial recently may be excluded. This is to avoid any interference between different study treatments.
  • Patients with a history of allergic reactions to the study drugs or similar medications are not eligible. This is to prevent any potential allergic reactions during the study.
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Investigated drugs

  • Venetoclax

    is a medication used in this trial to help treat patients with a type of blood disorder called higher-risk myelodysplastic syndrome (MDS). It works by targeting and blocking a specific protein in cancer cells, which can help to stop the growth of these cells and potentially lead to their death. In this study, venetoclax is being tested to see if it can improve the overall survival of patients when used together with another medication.

  • Azacitidine

    is a standard treatment for higher-risk myelodysplastic syndrome (MDS). It helps by interfering with the growth of abnormal blood cells and promoting the production of normal blood cells. In this trial, azacitidine is used as a part of the standard care, and researchers are studying its effects when combined with venetoclax to see if this combination can provide better outcomes for patients.

What is already known about the treatment

  • Venetoclax

    This medication is administered orally and is currently being studied in combination with azacitidine for the treatment of higher-risk myelodysplastic syndrome (MDS). It is in the clinical trial phase, with ongoing research to evaluate its safety and effectiveness. Venetoclax is primarily used for its potential to improve overall survival in patients with this condition. It works by inhibiting a protein called BCL-2, which helps cancer cells survive, thereby promoting their death. Venetoclax is classified as a BCL-2 inhibitor.

  • Azacitidine

    This medication is administered via injection and is a standard treatment for higher-risk myelodysplastic syndrome (MDS). It is well-established in medical practice and literature as a treatment option for this condition. Azacitidine is used to help improve blood cell counts and reduce the need for blood transfusions in patients. It works by incorporating into the DNA of cancer cells, disrupting their growth and leading to cell death. Azacitidine is classified as a DNA methyltransferase inhibitor.

Investigated diseases

Higher-Risk Myelodysplastic Syndrome – This is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when something goes wrong in the bone marrow, where blood cells are produced. In higher-risk cases, the condition can progress more rapidly and may lead to severe anemia, infections, or bleeding due to low blood cell counts. Over time, the bone marrow may become more dysfunctional, leading to a higher risk of developing acute myeloid leukemia. The disease is characterized by an increased number of immature blood cells, known as blasts, in the bone marrow or blood. Symptoms often include fatigue, shortness of breath, and easy bruising or bleeding.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507153-16-00Protocol codeM15-954Estimated enrolment526 patientsSponsorAbbVie Deutschland GmbH & Co. KG

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