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Study on Treating Acute Myeloid Leukemia in Children and Adolescents Using CPX-351, Gemtuzumab Ozogamicin, and a Drug Combination

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on the treatment of Acute Myeloid Leukemia (AML) in children and adolescents. The study aims to explore the effectiveness of different treatment approaches for this type of cancer, which affects the blood and bone marrow. The trial involves several medications, including Vyxeos Liposomal (containing cytarabine and daunorubicin), Gemtuzumab Ozogamicin, Melphalan, Cyclophosphamide, Idarubicin Hydrochloride, Prednisolone, Busulfan, Fludarabine, Thiopeta, Treosulfan, Etoposide, Methotrexate, and Mitoxantrone Hydrochloride. These medications are used in various combinations to assess their impact on the disease.

The purpose of the study is to determine if these treatments can improve outcomes for patients with AML. The trial is divided into different groups, each focusing on specific treatment strategies. For example, one group will evaluate the use of CPX-351 (another name for Vyxeos Liposomal) to see if it can improve event-free survival, which means the time patients remain free from certain complications or events related to the disease. Another group will assess the initial effectiveness of Gemtuzumab Ozogamicin when added to standard chemotherapy for children whose AML has returned or is resistant to treatment. Additionally, the study will compare different conditioning regimens, which are treatments given before a stem cell transplant, to see which is more effective in preventing complications like graft-versus-host disease, a condition where the donor cells attack the recipient's body.

Participants in the study will receive these treatments over a period of time, with regular monitoring to track their progress and any side effects. The trial will also collect samples for further biological and toxicity studies to better understand the effects of these treatments. The study is open-label, meaning both the researchers and participants know which treatments are being administered. The trial is expected to continue until the end of 2029, with the goal of improving treatment strategies for children and adolescents with AML.

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

    Initial assessment

    Upon joining the clinical trial, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a physical examination.

    Blood tests and other diagnostic procedures may be performed to ensure the patient meets the criteria for participation.

  2. Step 2

    Treatment group assignment

    Patients are assigned to one of the treatment groups based on specific criteria related to their condition.

    The assignment determines the specific treatment regimen the patient will follow during the trial.

  3. Step 3

    Treatment administration

    Patients receive medications as part of their treatment plan. The medications include cytarabine, daunorubicin, melphalan, anhydrous cyclophosphamide, idarubicin hydrochloride, prednisolone, busulfan, gemtuzumab ozogamicin, thiotepa, treosulfan, etoposide, methotrexate, and mitoxantrone hydrochloride.

    The medications are administered through various routes such as intravenous (IV) infusion, injection, or intrathecal (into the spinal canal).

    The frequency and dosage of each medication depend on the specific treatment protocol assigned to the patient.

  4. Step 4

    Monitoring and follow-up

    Regular monitoring is conducted to assess the patient's response to treatment and to identify any side effects.

    Follow-up visits include physical examinations, blood tests, and other necessary evaluations to ensure the patient's safety and the effectiveness of the treatment.

  5. Step 5

    Completion of treatment

    Upon completion of the treatment regimen, a final assessment is conducted to evaluate the overall outcome.

    Patients may be asked to participate in additional follow-up visits to monitor long-term effects and gather data for the study.

Who can join the trial?

12 criteria

  • All participants: Must understand and voluntarily provide written permission from a parent or legal representative before any study-related assessments or procedures.
  • Group C: Must have **Acute Myeloid Leukemia (AML)** and need a first allogeneic **Hematopoietic Stem Cell Transplant (HSCT)** from a donor who is a close genetic match (at least 9 out of 10 match).
  • Group C: Must be between 1 month (28 days) and less than 18 years old at diagnosis; up to 21 years old at the time of HSCT.
  • Group C: Must meet criteria for allogeneic HSCT, which includes being in complete remission from AML and having a matched sibling or unrelated donor.
  • All participants: Must be able to follow the study visit schedule and other study requirements.
  • All female participants who can have children: Must have a negative pregnancy test within 10 days before starting treatment.
  • Group A: Must have a diagnosis of **de-novo AML** according to the 2016 WHO classification.
  • Group A: Must have **acute leukemia of ambiguous lineage**, which includes types like acute undifferentiated leukemia and others as per the 2016 WHO classification.
  • Group A: Must be children and adolescents under 18 years old at the start of initial chemotherapy.
  • Group A: Must agree that vaccination with live vaccines is not allowed while participating in the trial.
  • Group B: Must have first relapsed or primary refractory AML, including relapse after a stem cell transplant.
  • Group B: Must be children and adolescents under 18 years old at the start of initial chemotherapy and under 21 years old at the start of treatment for relapsed AML.

Who cannot join the trial?

5 criteria

  • Patients with a history of another type of cancer, known as a second malignant neoplasm, cannot participate.
  • Patients who have experienced severe liver problems, specifically VOD Grade 3 or 4 (a condition affecting the liver), are excluded.
  • Patients who have had severe forms of GvHD (Graft versus Host Disease), which is a complication that can occur after a transplant, are not eligible. This includes Acute GvHD grade 3-4 and Chronic GvHD (moderate and severe).
  • Patients who have had a clinical or molecular relapse, meaning their disease has returned or worsened, cannot join the study.
  • Patients who have passed away from any cause are obviously not eligible.
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Investigated drugs

  • CPX-351

    is a special type of chemotherapy used to treat a form of blood cancer called acute myeloid leukemia (AML). It is designed to deliver two chemotherapy drugs directly to the cancer cells in a way that helps them work better together. This medication is used to see if it can help patients live longer without the cancer coming back.

  • Gemtuzumab Ozogamicin

    is a medication that combines an antibody with a chemotherapy drug. The antibody part helps the medication find and attach to the cancer cells, and the chemotherapy part helps kill them. This medication is being tested to see if it can help children whose AML has come back or hasn't responded to other treatments.

  • Treosulfan

    is a type of chemotherapy used as part of a conditioning regimen before a bone marrow transplant. It helps prepare the body to receive new, healthy stem cells by killing cancer cells and making space in the bone marrow. This study is looking at how well Treosulfan works compared to another regimen in preventing complications after the transplant.

  • BuCyMel

    is a combination of three chemotherapy drugs used together as a conditioning regimen before a bone marrow transplant. This combination helps destroy cancer cells and prepare the body for new stem cells. The study is comparing this regimen to another to see which one is better at preventing complications after the transplant.

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) in children and adolescents. The main therapeutic indication for CPX-351 is to improve event-free survival in patients with newly diagnosed AML. At the molecular level, CPX-351 works by delivering a combination of two chemotherapy drugs, cytarabine and daunorubicin, 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.

  • Gemtuzumab Ozogamicin

    Gemtuzumab Ozogamicin is administered as an intravenous infusion, allowing the medication to enter the bloodstream directly. It is being evaluated in clinical trials for its potential to treat relapsed or refractory acute myeloid leukemia (AML) in children. The main therapeutic indication is to assess its initial effectiveness when added to standard chemotherapy. Gemtuzumab Ozogamicin works by targeting a specific protein on the surface of leukemia cells, delivering a toxic substance that helps destroy these cancerous cells. It is classified as an antibody-drug conjugate, which combines an antibody with a chemotherapy drug to target cancer cells more precisely.

  • Treosulfan

    Treosulfan is administered intravenously, meaning it is given directly into the bloodstream through a vein. It is being studied in clinical trials to compare its effectiveness as part of a conditioning regimen for patients undergoing treatment for acute myeloid leukemia (AML). The main therapeutic indication is to evaluate its impact on reducing complications and improving outcomes compared to other conditioning regimens. Treosulfan works by interfering with the DNA of cancer cells, preventing them from growing and dividing. It is classified as an alkylating agent, a type of chemotherapy that damages the DNA of cancer cells to stop their growth.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia (AML) 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. AML can develop quickly and requires prompt medical attention. The disease is more common in adults but can also occur in children. It is distinct from other types of leukemia due to its specific cellular characteristics and rapid progression.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-500783-35-00Protocol codeAIEOP-BFM AML 2020Estimated enrolment1 091 patientsSponsorGPOH gGmbH

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