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Study Comparing CPX-351 and Chemotherapy for Patients with New Intermediate or High-Risk 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 is comparing two different treatments for patients with newly diagnosed AML that is considered to be of intermediate or high risk based on genetic factors. One treatment being tested is a medication called CPX-351, which is a combination of two drugs, cytarabine and daunorubicin, delivered in a special form called liposomal. The other treatment is a more traditional intensive chemotherapy approach using a combination of idarubicin and cytarabine.

The purpose of the study is to see if CPX-351 can help more patients achieve a deep remission, which means the cancer is significantly reduced or undetectable, compared to the traditional chemotherapy. Patients in the study will be randomly assigned to receive either CPX-351 or the traditional chemotherapy. The study will monitor the patients' response to the treatment, focusing on how well the cancer is controlled and how the patients' bodies handle the treatment. The study will also look at the quality of life of the patients during the treatment.

Throughout the study, patients will receive their assigned treatment through an intravenous infusion, which means the medication is given directly into a vein. The study will last for several months, with regular check-ups and assessments to track the progress of the treatment. The goal is to gather information that could help improve treatment options for people with AML in the future.

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, you will be randomly assigned to one of two treatment groups. This means you will either receive the experimental treatment or the standard treatment. This assignment is done by chance, like flipping a coin.

  2. Step 2

    Treatment induction

    If you are in the experimental group, you will receive a medication called CPX-351. This is a combination of two drugs, cytarabine and daunorubicin, given through a vein (intravenous).

    If you are in the standard treatment group, you will receive a combination of idarubicin and cytarabine, also given intravenously. This is known as the 3+7 regimen, which means you will receive idarubicin for 3 days and cytarabine for 7 days.

  3. Step 3

    Monitoring and assessments

    Throughout the treatment, regular assessments will be conducted to monitor your response. This includes checking for complete remission (CR) or complete remission with incomplete hematologic recovery (CRi).

    These assessments involve taking samples from your bone marrow to measure the level of disease using specific methods.

  4. Step 4

    Consolidation therapy

    After the initial treatment, you may receive additional therapy called consolidation therapy. This is to help maintain remission and prevent the disease from returning.

    The timing and type of consolidation therapy will depend on your response to the initial treatment.

  5. Step 5

    Follow-up

    You will have regular follow-up visits to monitor your health and any long-term effects of the treatment. These visits are important to ensure your well-being and to address any concerns you may have.

Who can join the trial?

9 criteria

  • You must have been diagnosed with Acute Myeloid Leukemia (AML) for the first time (this is called de novo AML).
  • You must not have a specific type of genetic change in your leukemia cells known as MRC-defining cytogenetic lesion.
  • You must not have certain genetic changes in your leukemia cells, specifically t(15;17), t(8;21), inv(16), or t(16;16).
  • You must not have a mutation in the NPM1 gene.
  • You must not have a type of AML with mutations in the FLT3 gene (either FLT3 ITD or TKD).
  • You must not have received any previous treatment for AML, except for a short course of a medication called hydroxyurea if you had a high white blood cell count or symptoms from the tumor.
  • You must be 50 years old or older.
  • Your overall health and ability to carry out daily activities must be at a level of 2 or less on the ECOG performance status scale, which measures how the disease affects your daily living abilities.
  • Both men and women can participate.

Who cannot join the trial?

8 criteria

  • Patients with any other type of cancer that is not Acute Myeloid Leukemia.
  • Patients who have received any other treatment for Acute Myeloid Leukemia before the trial.
  • Patients with severe heart problems.
  • Patients with uncontrolled infections.
  • Patients who are pregnant or breastfeeding.
  • Patients with a history of severe allergic reactions to the drugs used in the trial.
  • Patients with any other serious medical condition that might interfere with the trial.
  • Patients who are unable to follow the trial procedures.
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Investigated drugs

  • CPX-351

    is a special type of chemotherapy treatment used in this clinical trial. It is designed to help treat a type of blood cancer called acute myeloid leukemia (AML). CPX-351 is a combination of two chemotherapy drugs that are packaged together in a way that helps them work better against cancer cells. The goal of using CPX-351 is to help patients achieve a deep remission, which means reducing the number of cancer cells in the body to very low levels.

  • Idarubicin

    is a chemotherapy drug used in this trial as part of the control treatment. It works by interfering with the DNA inside cancer cells, which stops them from growing and dividing. Idarubicin is used to help treat AML by killing cancer cells and reducing the number of cancer cells in the body.

  • Cytarabine

    is another chemotherapy drug used in the control treatment of this trial. It works by stopping cancer cells from making and repairing DNA, which is essential for their growth and survival. Cytarabine is used alongside idarubicin to help treat AML by targeting and destroying cancer cells, aiming to achieve remission in patients.

What is already known about the treatment

CPX-351 – CPX-351 is administered as an intravenous infusion, meaning it is given directly into the bloodstream through a vein. It is currently being studied in clinical trials for its effectiveness in treating Acute Myeloid Leukemia (AML), a type of cancer that affects the blood and bone marrow. The main therapeutic use of CPX-351 is to help patients achieve remission, which means reducing the number of cancer cells to very low levels. At the molecular level, CPX-351 works by delivering two chemotherapy drugs, daunorubicin and cytarabine, in a specific ratio that enhances their ability to kill cancer cells. It is classified as an antineoplastic agent, which is a type of medication used to treat cancer.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia is a type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells. These abnormal cells accumulate in the bone marrow, interfering with the production of normal blood cells. As the disease progresses, it can lead to symptoms such as fatigue, frequent infections, and easy bruising or bleeding due to the lack of healthy blood cells. The disease can advance quickly, requiring prompt medical attention. It is most common in adults and can vary in its specific genetic and molecular characteristics. The progression of the disease can be monitored through various methods, including the assessment of minimal residual disease in the bone marrow.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-500295-60-00Protocol code21-PP-26Estimated enrolment248 patientsSponsorCentre Hospitalier Universitaire De Nice

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