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Study of Cytarabine, Daunorubicin, and Clofarabine for Children with Relapsed or Refractory Acute Myeloid Leukemia (AML)

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) in children. The study is specifically for children whose AML has returned after treatment or has not responded to previous treatments. The trial will test a combination of two treatments: Vyxeos, which contains the active substances cytarabine and daunorubicin, and Clofarabine. These medications are given as a solution through a vein, which is known as an infusion.

The purpose of the study is to find the best dose of the combination of Vyxeos and Clofarabine for children with relapsed or refractory AML. During the study, participants will receive the treatment and be monitored for any side effects and how well the treatment works against the cancer. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the actual treatment.

The study will also look at how safe the treatment is, how well it is tolerated by the participants, and how it affects the cancer. Researchers will gather information on the overall survival of the participants, the time they remain free from cancer returning, and the number of participants who can undergo a bone marrow transplant after the treatment. The study aims to provide valuable information that could help improve treatment options for children with AML in the future.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    The initial assessment involves a complete work-up within 7 days prior to joining the study. This includes a bone marrow aspiration and a lumbar puncture, which is a procedure to collect fluid from the spine. No medication is administered during this procedure.

  2. Step 2

    Treatment phase

    The treatment involves the administration of two medications: Vyxeos Liposomal and Clofarabine. Both are given as solutions for infusion, which means they are delivered directly into the bloodstream through a vein.

    Vyxeos Liposomal contains two active substances, cytarabine and daunorubicin, and is administered intravenously. The specific dosage and frequency will be determined by the study team based on individual needs.

    Clofarabine is also administered intravenously. The dosage and frequency will be adjusted according to the study protocol and individual response.

  3. Step 3

    Monitoring and evaluation

    Throughout the treatment, regular monitoring will occur to assess the body's response to the medications. This includes checking for any dose-limiting toxicities, which are side effects that prevent an increase in dosage.

    Safety and tolerability will be evaluated by observing any adverse effects and significant changes in laboratory test results. The study will also measure the anti-leukemic activity of the treatment.

  4. Step 4

    Follow-up

    After completing the treatment phase, follow-up assessments will be conducted to evaluate overall survival and relapse-free survival. This involves tracking the patient's health status and any signs of the disease returning.

    Additional tests may be performed to understand the relationship between the treatment response and specific biological markers.

Who can join the trial?

12 criteria

  • The patient must have had at least a second relapse of **AML**. **AML** stands for **Acute Myeloid Leukemia**, which is a type of blood cancer.
  • The patient has **refractory AML**, meaning the cancer did not respond to standard treatment and there are 20% or more cancer cells in the bone marrow.
  • The patient experienced an early first relapse of **AML**, which means the cancer returned within one year of the initial diagnosis.
  • The patient has had any relapse of **AML** after a previous **allogenic HSCT**. **Allogenic HSCT** is a type of stem cell transplant from a donor.
  • The patient has had any relapse of **AML** with high-risk **cytogenetic characteristics**. **Cytogenetic characteristics** refer to specific changes in the chromosomes of the cancer cells.
  • The patient must complete an initial work-up within 7 days before joining the study. This includes a bone marrow aspiration and a lumbar puncture, which are tests to check the bone marrow and spinal fluid.
  • For patients under 16 years old, a **Lansky play score** of 60 or higher is required. This score measures the child's ability to play and perform daily activities.
  • For patients 16 years or older, a **Karnofsky performance status** of 60 or higher is needed. This status measures the patient's ability to carry out daily activities.
  • The patient must have a life expectancy of more than 6 weeks.
  • The patient must have a **calculated GFR** of 70 mL/min/1.73 m² or higher. **GFR** stands for **Glomerular Filtration Rate**, which measures kidney function.
  • The patient's liver function must be adequate, with total serum **bilirubin** of 3 mg/dL or less, and **AST** and **ALT** levels of 200 U/L or less. **Bilirubin**, **AST**, and **ALT** are substances measured to check liver health.
  • The patient must have adequate heart function, with a **shortening fraction** of 28% or higher or an **ejection fraction** of 50% or higher. These are measurements of how well the heart is pumping blood.
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Investigated drugs

  • Vyxeos

    This medication is a combination of two drugs, daunorubicin and cytarabine, in a special liposomal form. It is used to treat certain types of blood cancer, specifically acute myeloid leukemia (AML). The liposomal form helps deliver the drugs directly to the cancer cells, potentially improving their effectiveness and reducing side effects.

  • Clofarabine

    This is a chemotherapy drug used to treat certain types of leukemia, including acute myeloid leukemia (AML). It works by interfering with the growth and multiplication of cancer cells, helping to slow down or stop the progression of the disease.

What is already known about the treatment

  • Vyxeos

    This medication is administered intravenously and is currently being studied in clinical trials for its effectiveness in treating relapsed or refractory acute myeloid leukemia (AML) in children. Vyxeos is a combination of two chemotherapy drugs, daunorubicin and cytarabine, encapsulated in a liposomal formulation to enhance delivery to cancer cells. It works by interfering with the DNA of cancer cells, preventing them from growing and dividing. Vyxeos is classified as an antineoplastic agent, specifically a combination chemotherapy drug.

  • Clofarabine

    This medication is also administered intravenously and is being evaluated in combination with Vyxeos for treating relapsed or refractory AML in children. Clofarabine is a chemotherapy drug that targets rapidly dividing cells, such as cancer cells, by inhibiting DNA synthesis and repair. It is classified as an antimetabolite, which disrupts the growth of cancer cells by mimicking the building blocks of DNA.

Investigated diseases

Acute Myeloid Leukemia (AML) – Acute Myeloid Leukemia is a type of cancer that starts in the blood-forming cells of the bone marrow and quickly progresses to affect the blood. It is characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. This leads to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. As the disease progresses, it can spread to other parts of the body, including the lymph nodes, liver, spleen, and central nervous system. AML is more common in adults but can also occur in children. The progression of the disease can vary, with some cases advancing rapidly while others may progress more slowly.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-508050-26-00Protocol codeSP_MH20VYXEstimated enrolment25 patientsSponsorPrincess Maxima Center For Pediatric Oncology

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