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Pharmacokinetic, Safety, and Efficacy Evaluation of ASTX727 and Venetoclax in Adult Patients with Acute Myeloid Leukemia

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 a type of blood cancer called Acute Myeloid Leukemia (AML). The study will explore the effects of a treatment combination involving two medications: ASTX727 and Venetoclax. ASTX727 is a combination of two substances, Decitabine and Cedazuridine, which are taken in tablet form. Venetoclax is also taken as a tablet and is used to help treat certain types of blood cancers.

The purpose of this study is to understand how these medications work together in the body and to assess their safety and effectiveness in treating AML. Participants in the study will take these medications over a period of time, and researchers will monitor how the drugs interact with each other and how well they work in treating the cancer. The study will also look at any side effects that may occur during the treatment.

Participants will be involved in different phases of the study, where the main focus will be on how the body processes the medications and the overall response to the treatment. The study aims to provide valuable information on the potential benefits and risks of using ASTX727 and Venetoclax together for treating AML.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>medication administration</b>

    The treatment involves taking two different medications by mouth: astx727 (which contains decitabine and cedazuridine) and venclyxto (which contains venetoclax).

    The dosage for astx727 is 999 mg in tablet form.

    The dosage for venclyxto is 400 mg in film-coated tablet form.

  2. Step 2

    <b>study evaluation phases</b>

    The process is divided into different stages to monitor how the body processes the drugs and how effective the combination is for acute myeloid leukemia (a type of blood cancer).

    During phase 1, the focus is on how astx727 affects the levels of venetoclax in the body.

    In phase 2 part a, the clinical response to the combined therapy is evaluated, alongside the study of how the drugs interact with each other.

    In phase 2 part b, the clinical response to the combined therapy is continued to be evaluated.

  3. Step 3

    <b>monitoring and cycles</b>

    The treatment is organized into cycles.

    During the second cycle, specific measurements are taken on day 5 while taking both astx727 and venetoclax, and on day 15 when astx727 is not taken, to compare the levels of venetoclax in the blood.

Who can join the trial?

11 criteria

  • You must be at least 18 years old.
  • You must have a confirmed diagnosis of Acute Myeloid Leukemia (AML), which is a type of cancer that affects the blood and bone marrow, using the official standards set by the World Health Organization (WHO).
  • It is expected that you will live for at least 3 months.
  • You must not be able to receive intensive induction chemotherapy, which is a very strong type of treatment used to kill cancer cells quickly. This applies if you are 75 years of age or older, or if you are between 18 and 74 years old and have certain health conditions.
  • You must have a severe cardiac disorder, which means a serious heart problem, such as congestive heart failure (where the heart cannot pump blood well enough) or stable angina (chest pain caused by reduced blood flow to the heart).
  • You must have a severe pulmonary disorder, which means a serious lung problem that affects how well you can breathe or how much oxygen your lungs can transfer to your blood.
  • You must have a creatinine clearance level between 30 mL/min and less than 45 mL/min, which is a measure used to check how well your kidneys are filtering waste from your blood.
  • You must have moderate hepatic impairment, which means your liver is not working as well as it should, specifically measured by total bilirubin levels (a substance made when old red blood cells break down) that are slightly above the normal range.
  • You must have an ECOG Performance Status of 0 to 2 for the first part of the study, or 0 to 3 for the second part. This is a scale used by doctors to measure how well you can perform daily activities and your overall level of physical functioning.
  • You can be male or female.
  • You must be able to provide informed consent, which means you understand all the details of the study and agree to follow the rules and requirements to participate.

Who cannot join the trial?

21 criteria

  • A history of myeloproliferative neoplasms, which are a group of diseases where the bone marrow makes too many abnormal blood cells, such as myelofibrosis, essential thrombocythemia, polycythemia vera, or specific types of leukemia.
  • Having a significant history of serious problems with the kidneys, nervous system, mental health, hormones, metabolism, immune system, liver, heart, or lungs.
  • Any other medical condition or a known hypersensitivity (an extreme allergic reaction) to the study drugs that might make participating unsafe.
  • Having a serious systemic infection (an infection spreading through the body) caused by a virus, bacteria, or fungus that requires medical treatment.
  • A history of other malignancies (cancerous growths), except for certain skin cancers or early-stage cancers that have been successfully treated.
  • A white blood cell count (the cells that help fight infection) that is higher than 25,000 per microliter.
  • Current or previous treatment with hypomethylating agents (medicines used to treat blood disorders), venetoclax, CAR-T cell therapy (a type of immune system treatment), or other experimental drugs for blood disorders.
  • The inability to stop taking certain antifungal medicines or other drugs that affect the CYP3A enzyme (a protein in the liver that helps break down medicines) for a specific amount of time before starting the study.
  • The inability to stop taking medicines that are strong inhibitors (medicines that slow down how the body processes other drugs) or to avoid inducers (medicines that speed up how the body processes other drugs) of certain liver enzymes.
  • Being currently enrolled in another research study that involves medical treatments or specific procedures.
  • A known allergy to decitabine, cedazuridine, venetoclax, or any of the inactive ingredients used to make these medicines.
  • Having specific karyotype abnormalities, which are changes in the structure or number of chromosomes (the tiny structures that hold your DNA) in your cells.
  • Having a significant mental illness or active addiction to alcohol or other substances that might make it difficult to follow the study rules.
  • Eating grapefruit, grapefruit products, Seville oranges, or starfruit within 7 days before the first day of the study.
  • The cancer being active in the central nervous system (the brain and spinal cord).
  • A known infection of the human immunodeficiency virus (HIV).
  • A known active hepatitis B or hepatitis C infection (infections that affect the liver).
  • Severe hepatic impairment, which means the liver is not working properly, as measured by high levels of bilirubin or liver enzymes like AST, SGOT, ALT, or SGPT in the blood.
  • Severe renal impairment, which means the kidneys are not filtering blood effectively, as measured by a low creatinine clearance or glomerular filtration rate.
  • Having a malabsorption syndrome (a condition where the body cannot properly absorb nutrients from food) or any condition that prevents taking medicine by mouth.
  • Having significant cardiovascular disability, specifically a condition where physical activity causes fatigue, heart palpitations, shortness of breath, or chest pain.
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Investigated drugs

  • ASTX727

    is an oral tablet medication used in this study to evaluate how it works when combined with other treatments for acute myeloid leukemia.

  • Venetoclax

    is an oral medication used in this study to evaluate its effectiveness and how it interacts with other drugs in treating acute myeloid leukemia.

What is already known about the treatment

Decitabine - This medication is taken as an oral tablet and is currently used as an approved treatment for certain types of blood cancers. It works by attaching to the DNA of cancer cells to turn off the genes that allow them to grow uncontrollably. As a type of DNA methyltransferase inhibitor, it helps restart the body's natural ability to fight these cells. Cedazuridine - This substance is administered orally in tablet form and is used in combination therapies to help other drugs work more effectively. It acts by blocking a specific enzyme in the body that breaks down certain cancer medications, thereby keeping the treatment levels higher for longer. It is classified as an enzyme inhibitor used to enhance the performance of other therapeutic agents. Venetoclax - This medication is taken orally in the form of film-coated tablets and is an approved treatment for specific types of leukemia. It works by targeting and blocking a specific protein that cancer cells use to avoid programmed cell death, effectively forcing the cancer cells to die. It belongs to a class of drugs known as BCL-2 inhibitors.

Investigated diseases

Acute Myeloid Leukemia - This is a type of cancer that affects the blood and bone marrow. It occurs when the body produces an abnormal number of immature white blood cells, often called myeloblasts. These abnormal cells do not function correctly and crowd out healthy blood cells. As the disease progresses, the buildup of these cells can interfere with the production of red blood cells, platelets, and normal white blood cells. This imbalance affects the overall ability of the blood to perform its regular duties.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-516294-78-00Protocol codeASTX727-07Estimated enrolment160 patientsSponsorTaiho Oncology Inc.

sourced from the EU Clinical Trials Register and site verification

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