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Study of Axitinib and Etoposide for Children and Young Adults with Refractory or Relapsing Medulloblastoma and Ependymoma

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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 types of brain tumors: medulloblastoma and ependymoma. These are serious conditions that can occur in children and young adults. The study is testing a combination of two medications: axitinib, known by its brand name Inlyta, and etoposide. Axitinib is a medication that helps to block certain proteins that can promote cancer growth, while etoposide is a chemotherapy drug that helps to stop cancer cells from dividing and growing.

The purpose of this study is to find out how well these medications work together in treating patients whose tumors have not responded to standard treatments or have returned after treatment. The study will be conducted in two stages. In the first stage, researchers will determine the best dose of axitinib to use with a fixed dose of etoposide. In the second stage, they will evaluate how effective this combination is in preventing the tumors from growing or spreading further.

Participants in the study will receive the medications and be monitored over time to see how their tumors respond. The study will also look at the safety of the treatment and any side effects that may occur. This research aims to provide new insights into treatment options for children and young adults with these challenging types of brain tumors.

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, the patient will begin the first stage, known as the escalation part. This stage aims to determine the maximum tolerated dose of axitinib when combined with a fixed dose of oral metronomic etoposide.

  2. Step 2

    First stage: Dose escalation

    During this stage, the patient will receive axitinib and etoposide orally. The specific dosage and frequency will be adjusted to find the maximum tolerated dose of axitinib.

    The patient will be monitored for any dose-limiting toxicities over the first 28-day cycles.

  3. Step 3

    Second stage: Dose extension

    In the second stage, the patient will continue to receive axitinib at the dose determined during the first stage, along with the fixed dose of etoposide.

    The focus will be on estimating the effectiveness of the treatment in terms of progression-free survival, which is the time during which the patient's condition does not worsen.

  4. Step 4

    Monitoring and assessment

    Throughout the trial, the patient will undergo regular assessments to monitor tumor response and any adverse events. These assessments will be conducted according to specific criteria to ensure accurate evaluation.

    The patient's overall survival and progression-free survival will be tracked from the start of treatment until the end of the study or until any significant changes in health status occur.

  5. Step 5

    End of treatment

    At the conclusion of the treatment phase, the patient will have a final assessment to evaluate the overall response to the treatment.

    Any adverse events occurring during treatment or within 28 days after the end of treatment will be documented.

Who can join the trial?

19 criteria

  • Must have a confirmed diagnosis of either ependymoma or medulloblastoma. These are types of brain tumors.
  • Must be able to take medicines by mouth.
  • Must have proper organ function:
    • Blood health: Enough white blood cells and platelets, and hemoglobin levels can be supported by transfusion if needed.
    • Heart health: Heart must be working well, especially if the patient has had treatments that could affect the heart.
    • Kidney and liver health: Levels of certain substances in the blood must be within a safe range.
    • Must be able to follow the study schedule and manage any side effects.
    • If female and able to have children, must have a negative pregnancy test before starting the study.
    • If sexually active, must agree to use birth control during the study and for 6 months after.
    • Must be able to swallow capsules easily.
    • Must have written consent from parents or legal representatives, and agreement from the patient if they are old enough, before any study procedures begin.
    • Must be part of a social security system or have similar coverage as required locally.
    • Must have a specific test called methyloma classification done, or have material available for this test.
    • Must have a disease that is getting worse or not responding to standard treatments, or for which no effective standard treatment exists.
    • Must be between 4 and 25 years old.
    • Must weigh more than 20 kg (about 44 pounds).
    • Must have a tumor that can be measured according to specific guidelines.
    • Must have a performance status score of 70% or higher, which means they can do most daily activities. If unable to walk due to a stable condition, being in a wheelchair is acceptable.
    • Must have a life expectancy of at least 3 months.
    • Must not have any known allergies to the study treatment.

Who cannot join the trial?

6 criteria

  • Patients who have a different type of cancer than ependymoma or medulloblastoma cannot participate. Ependymoma and medulloblastoma are specific types of brain tumors.
  • Patients who are not children or young adults cannot participate. The study is for children and young adults only.
  • Patients who are not able to take the study medications as prescribed cannot participate.
  • Patients who have other serious health conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are not able to follow the study procedures or attend study visits cannot participate.
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Investigated drugs

  • Axitinib

    is a medication used in this trial to help treat children and young adults with certain types of brain tumors, specifically medulloblastoma and ependymoma, that have not responded to other treatments or have returned after treatment. It works by blocking certain proteins that help cancer cells grow, which may slow down or stop the growth of the tumor.

  • Etoposide

    is another medication used in this trial. It is given in a specific way called "oral metronomic," which means it is taken by mouth in small, regular doses over a period of time. This method aims to keep a steady level of the drug in the body to help fight the cancer cells more effectively. Etoposide works by interfering with the DNA of cancer cells, preventing them from dividing and growing.

What is already known about the treatment

  • Axitinib

    Axitinib is administered orally and is currently being studied in clinical trials for its effectiveness in treating certain types of brain tumors, specifically medulloblastoma and ependymoma, in children and young adults. It is classified as a tyrosine kinase inhibitor, which works by blocking specific proteins that promote the growth of cancer cells. This medication is being evaluated for its potential to improve progression-free survival in patients with these challenging conditions.

  • Etoposide

    Etoposide is taken orally in a metronomic dosing schedule, meaning it is given in low, regular doses. It is being investigated in combination with axitinib for its role in treating refractory or relapsing medulloblastoma and ependymoma. Etoposide is a chemotherapy drug that works by interfering with the DNA replication process in cancer cells, leading to cell death. This study aims to determine the maximum tolerated dose and assess the efficacy of this combination therapy.

Investigated diseases

  • Ependymoma

    Ependymoma is a type of tumor that arises from ependymal cells lining the ventricles of the brain and the central canal of the spinal cord. It can occur at any age but is more common in children. The tumor can cause symptoms by blocking the flow of cerebrospinal fluid, leading to increased pressure in the brain. As it grows, it may cause headaches, nausea, and balance problems. Ependymomas can vary in their growth rate, with some being slow-growing while others are more aggressive. The location of the tumor significantly influences the symptoms experienced by the patient.

  • Medulloblastoma

    Medulloblastoma is a fast-growing, high-grade tumor that originates in the cerebellum, the part of the brain that controls balance and coordination. It is most commonly diagnosed in children, but it can also occur in adults. The tumor can spread to other parts of the brain and spinal cord through the cerebrospinal fluid. Symptoms often include headaches, nausea, vomiting, and problems with coordination and balance. As the tumor progresses, it may lead to increased intracranial pressure and neurological deficits. Medulloblastomas are classified into different subtypes based on their genetic and molecular characteristics, which can influence their behavior and growth patterns.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-509585-38-00Protocol codeMEPENDAXEstimated enrolment40 patientsSponsorCentre Hospitalier Regional De Marseille

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