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Study of BGB-11417 and Azacitidine for Patients with Myeloid Malignancies (AML, MDS, MDS/MPN)

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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 treatments for myeloid malignancies, which include diseases like acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and myelodysplastic syndrome/myeloproliferative neoplasms (MDS/MPN). The study is testing a new treatment called BGB-11417, which is a type of medication known as a Bcl-2 inhibitor. This medication is being tested both on its own and in combination with another drug called azacitidine. Additionally, the study will look at how posaconazole, a medication used to prevent fungal infections, affects BGB-11417 when they are taken together.

The purpose of the study is to find out how safe and tolerable BGB-11417 is for patients with these diseases and to determine the best dose to use. The study will also evaluate how effective BGB-11417 is in treating these conditions. The trial is divided into different parts: the first part focuses on finding the right dose, the second part expands on safety, and the third part looks at how well the treatment works. During the study, participants will receive either BGB-11417 alone or in combination with azacitidine, and some will also receive posaconazole to see how it interacts with BGB-11417.

Participants in the study will be closely monitored to assess the safety of the treatments and to observe any side effects. The study aims to gather information on how the body processes BGB-11417 and how it affects the diseases being studied. This information will help researchers understand the potential benefits and risks of using BGB-11417 as a treatment option for myeloid malignancies.

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, participation begins with an initial assessment to confirm eligibility. This includes a review of medical history and current health status.

    Eligibility is based on a confirmed diagnosis of myeloid malignancies, such as acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).

  2. Step 2

    Part 1: dose regimen finding

    The first phase involves finding the appropriate dose of BGB-11417 in combination with azacitidine. This phase focuses on determining the safety and tolerability of the combination.

    During this phase, azacitidine is administered as a subcutaneous injection, while BGB-11417 is taken orally in the form of film-coated tablets.

  3. Step 3

    Part 2: safety expansion

    In the second phase, the focus is on expanding the safety evaluation of the combination treatment. The aim is to further assess the safety and tolerability of the selected dose regimen.

    Participants continue to receive azacitidine and BGB-11417 as previously described.

  4. Step 4

    Part 3: efficacy expansion

    The third phase evaluates the effectiveness of the treatment. This involves measuring response rates to the combination of BGB-11417 and azacitidine.

    This phase also examines the effect of posaconazole on the body's processing of BGB-11417 in a specific group of participants.

  5. Step 5

    Monitoring and follow-up

    Throughout the study, regular monitoring is conducted to assess health status and response to treatment. This includes routine blood tests and other necessary evaluations.

    Participants are observed for any side effects or adverse reactions to the medications.

Who can join the trial?

9 criteria

  • Must have a confirmed diagnosis of one of the following conditions according to the 2016 World Health Organization criteria:
    • Acute myeloid leukemia (AML), which is a type of cancer affecting the blood and bone marrow, but not acute promyelocytic leukemia.
    • Myelodysplastic syndrome (MDS), a group of disorders caused by poorly formed or dysfunctional blood cells.
    • Myelodysplastic syndrome/myeloproliferative neoplasms (MDS/MPN), which are conditions that have features of both MDS and MPN, another type of blood cancer.
    • Must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2, which is a scale used to assess how a patient's disease is progressing, including their level of functioning and ability to care for themselves.
    • Must have adequate organ function, which means:
      • Creatinine clearance of at least 50 milliliters per minute (mL/min), or between 30 and 49 mL/min for those in the unfit AML group. Creatinine clearance is a measure of kidney function.
      • Adequate liver function, meaning the liver is working well enough to handle the study treatment.
      • Must have a life expectancy of more than 12 weeks.
      • Must be able to comply with the requirements of the study, meaning they can follow the study procedures and visits.
      • Other specific criteria defined in the study protocol may also apply.

Who cannot join the trial?

10 criteria

  • Patients with other types of blood cancers that are not the focus of the study.
  • Patients who are not within the specified age range for the study.
  • Patients who are not able to safely tolerate the study medications.
  • Patients who are pregnant or breastfeeding.
  • Patients who have other serious health conditions that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent major surgery or injury.
  • Patients who have a history of allergic reactions to the study medications.
  • Patients who have an active infection that requires treatment.
  • Patients who have a history of drug or alcohol abuse.
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Investigated drugs

  • BGB-11417

    is a medication being studied for its potential to treat myeloid malignancies. It works by inhibiting a protein called Bcl-2, which can help cancer cells survive. The trial is exploring how safe and tolerable this medication is when used alone or in combination with another drug, azacitidine. Researchers are also trying to find the best dose to use in future studies.

  • Azacitidine

    is a medication used in combination with BGB-11417 in this trial. It is commonly used to treat certain types of blood cancers by helping to restore normal growth and function to blood cells. In this study, azacitidine is being used to see if it can enhance the effects of BGB-11417 in treating myeloid malignancies.

  • Posaconazole

    is an antifungal medication that is being studied for its interaction with BGB-11417. The trial aims to understand how posaconazole affects the way BGB-11417 is processed in the body when both drugs are taken together. However, this part of the study is not being conducted in Europe.

What is already known about the treatment

BGB-11417 – This medication is administered orally and is currently being studied in clinical trials for its potential use in treating myeloid malignancies, including acute myeloid leukemia and myelodysplastic syndromes. It is in the investigational stage, with ongoing research to determine its safety, tolerability, and optimal dosing. BGB-11417 works by inhibiting the Bcl-2 protein, which plays a role in cell survival, thereby promoting the death of cancer cells. It is classified as a Bcl-2 inhibitor and is being evaluated both as a monotherapy and in combination with azacitidine to enhance its therapeutic effects.

Investigated diseases

  • Acute Myeloid Leukemia (AML)

    A type of cancer that starts in the blood-forming cells of the bone marrow and quickly progresses to affect 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 may include fatigue, fever, frequent infections, and easy bruising or bleeding. As the disease progresses, it can spread to other parts of the body, including the lymph nodes, liver, and spleen. AML is more common in adults and can develop suddenly, requiring prompt medical attention.

  • Myelodysplastic Syndromes (MDS)

    A group of disorders caused by poorly formed or dysfunctional blood cells. These syndromes occur when the blood-forming cells in the bone marrow are damaged, leading to a shortage of one or more types of blood cells. Symptoms often include fatigue, shortness of breath, and increased risk of bleeding or infection. MDS can progress slowly or transform into a more aggressive form of leukemia. The condition is more common in older adults and can vary widely in severity.

  • Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN)

    A rare group of diseases that have features of both myelodysplastic syndromes and myeloproliferative neoplasms. These conditions are characterized by the abnormal growth and development of blood cells in the bone marrow. Patients may experience symptoms such as fatigue, fever, weight loss, and an enlarged spleen. The diseases can progress at different rates and may lead to complications such as anemia or increased risk of bleeding. MDS/MPN can affect individuals of any age but is more common in older adults.

Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-508881-14-00Protocol codeBGB-11417-103Estimated enrolment265 patientsSponsorBeOne Medicines AG

sourced from the EU Clinical Trials Register and site verification

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