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Study of Cytarabine and Daunorubicin for Treating Myeloid Leukemia in Children with Down Syndrome

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 Myeloid Leukemia in children with Down Syndrome. The treatment being tested is a medication called Vyxeos Liposomal, which contains two active substances: cytarabine and daunorubicin. These substances are used in a form that is given through an intravenous infusion, meaning it is administered directly into the bloodstream through a vein.

The purpose of the study is to evaluate the effectiveness of this treatment in achieving a specific outcome known as event-free survival, which refers to the time from diagnosis until a significant event occurs, such as a relapse or a new cancer. The study will involve children with Down Syndrome who have been diagnosed with Myeloid Leukemia. Participants will receive the treatment over a period of time, and their health will be monitored to see how well the treatment works and to observe any side effects.

Throughout the study, researchers will collect information on various aspects of the participants' health, including overall survival, which is the time from diagnosis to death from any cause, and disease-free survival, which is the time after treatment during which the patient remains free of disease. The study will also look at the early response rate to the treatment and any treatment-related side effects. The goal is to gather data that will help improve treatment options for children with this type of leukemia.

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 trial

    Upon joining the trial, you will be required to provide written permission from your parent or legal representative. This is necessary before any study-related assessments or procedures can begin.

    You must be able to adhere to the study visit schedule and other protocol requirements. This includes understanding that vaccination with live vaccines is not possible while participating in the trial.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm eligibility. This includes verifying that you have Down syndrome or mosaic, are within the specified age range, and meet other health criteria.

    Your performance status will be evaluated using the Lansky or Karnofsky performance score, which measures your ability to perform daily activities.

  3. Step 3

    Treatment phase

    The treatment involves receiving a medication called Vyxeos Liposomal, which contains the active substances cytarabine and daunorubicin. This medication is administered through an intravenous infusion, meaning it is given directly into a vein.

    The dosage, frequency, and duration of administration will be determined by the study protocol and your healthcare team.

  4. Step 4

    Monitoring and follow-up

    Throughout the trial, you will be closely monitored for any side effects or changes in your condition. This includes regular check-ups and tests to assess your response to the treatment.

    The primary goal is to achieve event-free survival (EFS), which means remaining free from certain negative events such as relapse or failure to achieve remission.

  5. Step 5

    Completion of trial

    At the end of the trial, a final assessment will be conducted to evaluate the overall outcomes and any long-term effects of the treatment.

    You will receive information about your health status and any necessary follow-up care.

Who can join the trial?

8 criteria

  • The child must have Down syndrome (also known as Trisomy 21) or a condition called mosaic Down syndrome.
  • The child must be older than 6 months and 4 years old or younger, with or without a specific genetic change called GATA1 mutation. If the child is older than 4 years but younger than 6 years, they must have the GATA1 mutation.
  • The child must have a specific type of blood cell appearance, known as FAB M0, M6, or M7, which are classifications of leukemia.
  • The child must have a performance score of at least 50 on either the Lansky or Karnofsky scale, which are measures of the child's ability to perform daily activities.
  • The child's parent or legal representative must understand and voluntarily provide written permission for the child to participate in the study, including the use of data and tumor samples, following international and local regulations.
  • The child and their family must be able to follow the study visit schedule and meet other study requirements.
  • The family must agree that the child will not receive any live vaccines while participating in the trial.
  • The child must have Myeloid Leukemia (ML) or Myelodysplastic Syndrome (MDS), as defined by the World Health Organization (WHO).

Who cannot join the trial?

4 criteria

  • Patients who do not have Myeloid Leukemia in children with Down Syndrome cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who do not meet the gender requirements for the study cannot participate.
  • Patients who are not considered part of a vulnerable population as defined by the study cannot participate.
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Investigated drugs

CPX-351 is a special type of chemotherapy used to treat a form of blood cancer called myeloid leukemia. It is designed to deliver two chemotherapy drugs, daunorubicin and cytarabine, in a way that helps them work better together. This combination is packaged in tiny fat-like particles called liposomes, which help the drugs stay in the body longer and target the cancer cells more effectively. The goal of using CPX-351 in this trial is to see if it can help children with Down syndrome who have myeloid leukemia achieve a similar success rate in treatment as seen in previous studies.

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 a Phase III clinical trial for its effectiveness in treating myeloid leukemia in children with Down syndrome. The main therapeutic indication for CPX-351 is to improve event-free survival (EFS) rates in these patients, aiming to match or exceed previous trial results. At the molecular level, CPX-351 works by delivering a combination of chemotherapy drugs, cytarabine and daunorubicin, in a specific ratio that enhances their ability to kill cancer cells. It is classified pharmacologically as a liposomal formulation of chemotherapy, designed to improve the delivery and effectiveness of the drugs while reducing side effects.

Investigated diseases

Myeloid Leukemia in Children with Down Syndrome – This disease is a type of blood cancer that occurs in children who have Down syndrome. It involves the rapid growth of abnormal white blood cells in the bone marrow, which can interfere with the production of normal blood cells. The disease typically progresses through several phases, starting with an accumulation of immature white blood cells. As it advances, it can lead to symptoms such as fatigue, easy bruising, and increased susceptibility to infections. The progression can vary, but it often involves a decrease in the body's ability to fight infections and control bleeding. Over time, the disease can affect various organs and systems in the body due to the spread of abnormal cells.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2022-501457-37-00Protocol codeML-DS2018Estimated enrolment238 patientsSponsorGPOH gGmbH

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