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Study of Emavusertib for Patients with Acute Myelogenous Leukemia or Myelodysplastic Syndrome

Verified siteInvestigationalNo 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 two blood-related diseases: Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndrome (MDS). These are conditions where the bone marrow, which is responsible for producing blood cells, does not function properly. The treatment being tested in this study is a medication called CA-4948, also known by its active ingredient emavusertib. This medication is taken orally in the form of a coated tablet.

The purpose of the study is to evaluate the safety and effectiveness of CA-4948 when used alone in patients with relapsed or refractory AML and higher-risk MDS. The study will be conducted in two phases. In the first phase, the focus is on determining the safest dose of the medication and understanding how well patients tolerate it. The second phase aims to assess the anti-cancer activity of CA-4948 in patients with specific genetic mutations, such as FMS-like tyrosine kinase-3 (FLT-3) mutations and spliceosome mutations like SF3B1 or U2AF1.

Participants in the study will receive the medication and be monitored for any side effects or changes in their condition. The study will also look at how the medication affects the body over time, including its concentration in the blood and how long it stays active. The trial is expected to continue until July 2025, allowing researchers to gather comprehensive data on the treatment's impact on these serious blood disorders.

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, the patient will provide written consent to participate and agree to comply with the study requirements.

    A series of initial tests will be conducted to confirm eligibility, including a bone marrow biopsy and blood tests to diagnose acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).

  2. Step 2

    Phase 1: dose escalation

    The patient will begin taking the medication CA-4948, which is a coated tablet taken orally. The dosage will be gradually increased to determine the maximum tolerated dose.

    During this phase, the patient's safety will be closely monitored through regular check-ups, including blood tests, electrocardiograms, and physical examinations.

  3. Step 3

    Phase 2a: cohort expansion

    Once the recommended dose is established, the patient will continue taking CA-4948 at this dose to assess its effectiveness against cancer.

    The patient's response to the treatment will be evaluated through regular medical assessments, including monitoring for any side effects and measuring the cancer's response.

  4. Step 4

    Ongoing monitoring

    Throughout the study, the patient will undergo regular health evaluations to ensure the treatment's safety and effectiveness.

    These evaluations will include blood tests, physical exams, and other necessary medical assessments.

  5. Step 5

    Completion of the study

    The study is expected to conclude by July 2025. Upon completion, the patient's overall health and response to the treatment will be thoroughly assessed.

    The patient will receive guidance on any further medical care needed after the study ends.

Who can join the trial?

16 criteria

  • Must be a male or female who is 18 years of age or older.
  • Must have a life expectancy of at least 3 months.
  • Must have an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 or 1, which means the patient is fully active or has some symptoms but can still carry out light work.
  • Must have a confirmed diagnosis of Acute Myelogenous Leukemia (AML) or higher-risk Myelodysplastic Syndrome (MDS) based on specific medical criteria.
  • Must have had no more than 2 previous treatments for cancer.
  • Must have acceptable organ function, which includes:
    • Kidney function with a creatinine clearance of at least 35 mL/min.
    • Liver enzymes (AST or ALT) no more than 2 times the upper limit of normal.
    • Total bilirubin no more than 1.5 times the upper limit of normal, or up to 3 times if the patient has Gilbert's syndrome.
    • White blood cell count no more than 25,000/µL.
    • Must have CPK levels below Grade 2, which means the levels are not significantly elevated.
    • If taking cholesterol-lowering medications that affect CPK levels, the medication should be adjusted if possible.
    • Must be able to swallow and keep down oral medications.
    • Women who can become pregnant must have a negative pregnancy test.
    • Women who can become pregnant and men with partners who can become pregnant must agree to use effective birth control during the study and for 180 days after the last dose of the study drug.
    • Must be willing to provide written consent to participate in the study and follow its requirements.
    • Must be willing to have bone marrow and blood samples taken during the study for diagnosis and evaluation.

Who cannot join the trial?

5 criteria

  • Patients with other types of cancer that are not Acute Myelogenous Leukemia (AML) or higher-risk Myelodysplastic Syndrome (MDS) cannot participate. AML is a type of blood cancer, and MDS is a condition where the bone marrow does not produce enough healthy blood cells.
  • Patients who have not experienced a return or worsening of their AML or MDS after treatment are not eligible. This means the study is for those whose condition has come back or has not improved with previous treatments.
  • Patients who do not have specific genetic mutations related to their AML or MDS, such as FMS-like tyrosine kinase-3 (FLT-3) mutations or spliceosome mutations of SF3B1 or U2AF1, cannot join. These are specific changes in the genes that can affect how the disease behaves.
  • Patients who are not within the specified age range for the study cannot participate. The study is open to adults, but specific age limits are set by the researchers.
  • Patients who are not able to follow the study procedures or who have other health conditions that might interfere with the study cannot participate. This ensures the safety of the participants and the accuracy of the study results.
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Investigated drugs

CA-4948 is an experimental medication being studied for its potential to treat certain types of blood cancers, specifically acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS). It is taken by mouth and works by targeting a specific protein involved in the growth and survival of cancer cells. The study aims to determine how safe and tolerable this medication is for patients, as well as to find the most effective dose. Researchers are also looking at how well it works in patients with specific genetic mutations related to these cancers.

What is already known about the treatment

CA-4948 – This medication is administered orally and is currently being studied in clinical trials for its potential use in treating acute myelogenous leukemia (AML) and higher-risk myelodysplastic syndrome (MDS). It is in the early phases of clinical research, focusing on its safety, tolerability, and effectiveness in patients with specific genetic mutations. CA-4948 works by inhibiting IRAK4, a protein involved in signaling pathways that can lead to cancer cell growth. It is classified as a kinase inhibitor, targeting molecular mechanisms that contribute to the progression of these blood disorders.

Investigated diseases

  • Acute Myelogenous Leukemia (AML)

    This is a type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells. These cells accumulate in the bone marrow and interfere with the production of normal blood cells. AML progresses quickly and can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. The disease is classified into different subtypes based on genetic and chromosomal abnormalities. It is more common in adults and can occur suddenly, requiring prompt medical attention.

  • Myelodysplastic Syndrome (MDS)

    This is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the blood-forming cells in the bone marrow are damaged, leading to a shortage of one or more types of blood cells. Higher-risk MDS can progress to acute myelogenous leukemia, a more aggressive form of blood cancer. Symptoms may include fatigue, shortness of breath, and increased risk of bleeding or infection. The condition is more prevalent in older adults and can vary in severity. MDS is often detected through routine blood tests showing abnormal blood counts.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-513313-13-00Protocol codeCA-4948-102Estimated enrolment366 patientsSponsorCuris Inc.

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