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Study on the Safety of Eganelisib Alone and with Cytarabine for Patients with Relapsed or Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndromes

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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 conditions: Acute Myeloid Leukemia (AML) and higher-risk Myelodysplastic Syndromes (HR-MDS). These are types of blood cancers that affect the bone marrow and blood cells. The study will explore the effects of a new treatment using a medication called Eganelisib, which is also known by its code name IPI-549. Eganelisib will be tested both on its own and in combination with another medication called Cytarabine, which is commonly used in treating these conditions.

The purpose of the study is to evaluate the safety and tolerability of Eganelisib, both as a standalone treatment and when combined with Cytarabine, in patients whose disease has returned or has not responded to previous treatments. The study will be conducted in two parts. In the first part, different doses of Eganelisib will be tested to find the most suitable dose for further research. In the second part, the study will look at how well the treatment works in controlling the disease.

Participants in the study will receive either Eganelisib alone or in combination with Cytarabine. The study will monitor the participants' health and any side effects they may experience. The goal is to determine the best dose of Eganelisib and to gather information on its potential benefits for patients with relapsed or refractory AML and HR-MDS. The study is expected to continue until early 2028, with recruitment starting in 2025.

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 visit and consent

    Upon joining the study, the first step involves an initial visit where the patient provides written informed consent. This consent confirms understanding of the study and agreement to participate.

    During this visit, the patient will undergo a series of tests to confirm eligibility, including blood tests and a review of medical history.

  2. Step 2

    Screening and baseline assessments

    The patient will undergo screening procedures to ensure they meet all study criteria. This includes a physical examination, blood tests, and possibly imaging studies.

    Baseline assessments are conducted to gather initial health data, which will be used for comparison throughout the study.

  3. Step 3

    Treatment phase - part 1

    In the first part of the treatment phase, the patient will receive eganelisib as a monotherapy. This involves taking eganelisib 5mg capsules or 30mg capsules orally, as determined by the study team.

    The dosage and frequency will be adjusted based on the patient's response and tolerance. Regular monitoring will occur to assess safety and any side effects.

  4. Step 4

    Treatment phase - combination therapy

    Following the monotherapy phase, the patient will receive a combination of eganelisib and cytarabine. Cytarabine is administered as an intravenous infusion.

    The combination therapy aims to evaluate the safety and effectiveness of the drugs when used together. The patient will be closely monitored for any adverse effects.

  5. Step 5

    Dose optimization

    In this phase, the study team will optimize the dosage of eganelisib and cytarabine based on the patient's response and tolerance.

    The goal is to determine the most effective dose with the least side effects. Regular assessments will continue to ensure patient safety.

  6. Step 6

    Follow-up and monitoring

    After completing the treatment phases, the patient will enter a follow-up period. This involves regular visits to monitor health status and any long-term effects of the treatment.

    The study team will conduct various tests and assessments to gather data on the treatment's impact over time.

Who can join the trial?

13 criteria

  • Provide written consent to participate in the study.
  • Both men and women of childbearing potential must agree to use effective birth control during the study and for a period after the study ends.
  • Women of childbearing potential must have a negative pregnancy test before starting the study and agree to ongoing pregnancy testing during the study.
  • Agree to use sun protection, such as sunscreen or protective clothing, during the study and for a period after the study ends.
  • Have a confirmed diagnosis of either:
    • Acute Myeloid Leukemia (AML) with at least 10% abnormal cells in the bone marrow, excluding a specific type called acute promyelocytic leukemia.
    • Higher-risk Myelodysplastic Syndromes (HR-MDS) with at least 10% abnormal cells in the bone marrow.
    • Have a disease that has returned or not responded to treatment, and no other effective treatments are available.
    • Be at least 18 years old.
    • Have a performance status of 0, 1, or 2 on the Eastern Cooperative Oncology Group (ECOG) scale, which measures daily living abilities.
    • Have adequate liver function, as shown by specific blood tests.
    • Have adequate kidney function, as shown by a creatinine clearance test of at least 30 L/min.
    • Have an acceptable blood clotting profile, as shown by specific blood tests.
    • Be willing and able to attend scheduled visits, follow the treatment plan, undergo laboratory tests, and participate in other study procedures, including bone marrow examinations.

Who cannot join the trial?

10 criteria

  • Patients with other types of cancer that are not the focus of this study.
  • Patients who have had a heart attack or stroke in the past 6 months.
  • Patients with uncontrolled high blood pressure.
  • Patients with active infections that require treatment.
  • Patients who are pregnant or breastfeeding.
  • Patients who have had an organ transplant.
  • Patients with severe liver or kidney disease.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had an allergic reaction to similar medications in the past.
  • Patients with a history of drug or alcohol abuse in the past year.
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Investigated drugs

  • Eganelisib

    is a medication being tested in this clinical trial to see if it is safe and well-tolerated by patients. It is being studied both on its own and in combination with another medication. The goal is to find out how well it works for people with a type of blood cancer called acute myeloid leukemia that has come back or has not responded to previous treatments.

  • Cytarabine

    is a medication that is often used to treat certain types of blood cancers, including acute myeloid leukemia. In this trial, it is being used in combination with eganelisib to see if the two medications together can help treat the cancer more effectively. The study aims to determine if this combination is safe and how well it works in patients whose cancer has returned or did not respond to other treatments.

What is already known about the treatment

Eganelisib – Eganelisib is administered orally and is currently being studied in clinical trials for its safety and effectiveness in treating relapsed or refractory acute myeloid leukemia (AML) and higher-risk myelodysplastic syndromes (HR-MDS). It is not yet widely used in medicine, as it is still under investigation. The main therapeutic use of Eganelisib is to help manage these specific types of blood cancers. At the molecular level, Eganelisib works by inhibiting a specific enzyme that plays a role in cancer cell survival and growth, potentially slowing down or stopping the progression of the disease. It is classified as a PI3K inhibitor, which is a type of targeted cancer therapy.

Investigated diseases

  • Acute Myeloid Leukemia

    Acute Myeloid Leukemia (AML) is a type of cancer that starts in the blood-forming cells of the bone marrow. It progresses rapidly, leading to the accumulation of immature white blood cells called myeloblasts. These cells crowd out normal blood cells, causing symptoms like fatigue, frequent infections, and easy bruising or bleeding. As the disease advances, it can spread to other parts of the body, including the lymph nodes, liver, and spleen. The rapid progression of AML requires prompt medical attention to manage its effects on the body.

  • Higher-Risk Myelodysplastic Syndromes

    Higher-Risk Myelodysplastic Syndromes (HR-MDS) are a group of disorders caused by poorly formed or dysfunctional blood cells. These syndromes lead to a decrease in the number of healthy blood cells, resulting in symptoms such as anemia, increased risk of infection, and bleeding issues. Over time, HR-MDS can progress to more severe forms of blood disorders, including acute myeloid leukemia. The progression involves the bone marrow producing more abnormal cells, which can further impair normal blood cell production. The condition requires careful monitoring to manage its impact on blood cell levels and overall health.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-518071-58-00Protocol codeSTLX-EGA-001Estimated enrolment116 patientsSponsorStelexis Biosciences Inc.

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