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Study on Ependymoma Treatment in Children, Adolescents, and Young Adults Using Cisplatin, Cyclophosphamide, Etoposide, 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 is focused on studying the treatment of ependymoma, a type of tumor that can occur in the brain or spine. The study involves children, adolescents, and young adults who have been newly diagnosed with this condition. The trial aims to explore different treatment strategies to improve outcomes for patients. The treatments being studied include a combination of chemotherapy drugs such as cisplatin, cyclophosphamide, etoposide, vincristine sulfate, methotrexate, and carboplatin. Additionally, the study will evaluate the use of sodium valproate, which is being tested for its potential to enhance the effects of chemotherapy.

The purpose of the study is to determine if certain treatment combinations can improve the chances of removing the tumor completely and increase the time patients remain free from disease progression. The study will also compare different chemotherapy schedules to see which is more effective for patients with tumors that could not be completely removed by surgery. For some patients, the study will assess the benefits of adding sodium valproate to the treatment plan, especially for those who cannot receive radiation therapy.

Participants in the study will undergo a series of treatments over several weeks, which may include surgery, chemotherapy, and possibly radiation therapy. The study will monitor the patients' progress through regular medical assessments, including MRI scans, to evaluate the effectiveness of the treatments. The trial is designed to gather information that could lead to better treatment options for ependymoma in the future.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and current health status.

    Eligibility criteria include being newly diagnosed with an intracranial or spinal ependymoma and having no previous chemotherapy or radiotherapy.

  2. Step 2

    Surgical resection

    Surgical resection is performed to remove the tumor. The extent of tumor removal is assessed through a centralized review of post-operative MRI scans.

  3. Step 3

    Stratum 1: chemotherapy and radiotherapy

    For patients with no residual measurable tumor, chemotherapy is administered for 16 weeks. The regimen includes cisplatin, cyclophosphamide, and etoposide (VEC+CDDP).

    Chemotherapy is followed by conformal radiotherapy.

  4. Step 4

    Stratum 2: chemotherapy schedules

    For patients with residual non-reoperable tumor, two chemotherapy schedules are compared: VEC or VEC+HD-MTX.

    The VEC regimen includes vincristine sulfate, etoposide, and cyclophosphamide. The VEC+HD-MTX regimen adds high-dose methotrexate.

  5. Step 5

    Stratum 3: chemotherapy with or without valproate

    For children unable to receive radiation therapy, chemotherapy is administered with or without valproate, a histone deacetylase inhibitor.

    The primary chemotherapy strategy is compared to the addition of valproate to evaluate progression-free survival.

  6. Step 6

    Follow-up and monitoring

    Regular follow-up visits are scheduled to monitor health status and treatment response.

    Progression-free survival and overall survival are evaluated as primary and secondary endpoints.

Who can join the trial?

24 criteria

  • The patient must live in one of the participating countries.
  • The patient should not have had any previous chemotherapy, except for steroids.
  • The patient should not have any other unrelated diseases, like kidney or blood diseases, that would prevent them from receiving chemotherapy.
  • The patient should not show any signs of infection.
  • The patient must have good bone marrow, liver, and kidney function.
  • The patient and/or their parents or legal guardians must be willing and able to attend scheduled visits, follow the treatment plan, and undergo laboratory tests and other study procedures.
  • Written informed consent must be provided by the patient and/or their parents or legal guardians.
  • For certain parts of the study, the patient must be older than 12 months and younger than 22 years at the time of joining the study.
  • The patient should not have any spread of the tumor to other parts of the body, as shown by spinal MRI and CSF (cerebrospinal fluid) tests.
  • The patient should not have any medical reasons that would prevent them from receiving radiotherapy or chemotherapy.
  • Post-menarchal females (females who have started menstruating) must not be pregnant or breastfeeding and must have a negative pregnancy test before starting the trial.
  • The patient must be younger than 22 years old at the time of diagnosis.
  • Males and females who can have children must use effective birth control during their treatment and for 6 months after finishing their treatment.
  • For certain parts of the study, there should be no remaining measurable ependymoma (a type of tumor) based on a central review of MRI scans.
  • For other parts of the study, there should be a remaining tumor that cannot be operated on, as shown by a central review of MRI scans.
  • For another part of the study, children younger than 12 months or patients who cannot receive radiotherapy due to age, tumor location, or other decisions are eligible.
  • The patient should not have any medical reasons that would prevent them from receiving chemotherapy.
  • The patient must have a confirmed diagnosis of an intracranial or spinal ependymoma, which is a type of tumor, according to a local pathologist.
  • The patient must provide tumor tissue samples to a national referral pathology center.
  • Written informed consent must be provided for the collection of data and study biological samples.
  • The patient and/or their parents or legal guardians must be willing and able to follow the study schedule and agree to sign a written informed consent.
  • The patient must be part of a Social Security System in countries where this is required.
  • The patient must have a newly diagnosed intracranial ependymoma of WHO grade II-III, confirmed by a central pathological review.
  • The patient should not have had any previous radiotherapy.

Who cannot join the trial?

7 criteria

  • Patients who have not been newly diagnosed with an ependymoma, which is a type of tumor that can occur in the brain or spine.
  • Patients who have a type of ependymoma that is not included in the study, such as cellular, papillary, myxopapillary, clear-cell, tanycytic, or anaplastic ependymoma.
  • Patients who are not within the specified age range for the study.
  • Patients who are not able to undergo the required treatments, such as surgery, chemotherapy, or radiotherapy.
  • Patients who are unable to have a post-operative MRI, which is a type of scan used to look at the brain or spine after surgery.
  • Patients who are not able to receive valproate, a medication used in the study, if it is part of their treatment plan.
  • Patients who are not part of the vulnerable population selected for the study.
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Investigated drugs

  • VEC

    is a combination of three chemotherapy drugs used to treat cancer. It includes vincristine, etoposide, and cyclophosphamide. These medications work together to stop the growth of cancer cells by interfering with their ability to divide and multiply.

  • CDDP

    , also known as cisplatin, is a chemotherapy drug that contains platinum. It is used to treat various types of cancer by damaging the DNA of cancer cells, which prevents them from growing and dividing.

  • HD-MTX

    stands for high-dose methotrexate. Methotrexate is a chemotherapy drug that interferes with the growth of cancer cells. In high doses, it is used to treat certain types of cancer more aggressively.

  • Valproate

    is a medication that is primarily used to treat seizures and mood disorders. In this trial, it is used as a histone deacetylase inhibitor, which means it can help modify the expression of certain genes in cancer cells, potentially making them more sensitive to chemotherapy.

What is already known about the treatment

Valproate – Valproate is administered orally and is used in the treatment of various neurological conditions. In the context of this clinical trial, it is being evaluated as an additional treatment for children with ependymoma who cannot receive radiation therapy. Valproate works by inhibiting histone deacetylase, which can affect gene expression and potentially slow tumor growth. It is classified as an anticonvulsant and mood-stabilizing drug, and its use in this trial is experimental, aiming to improve progression-free survival when combined with chemotherapy.

Investigated diseases

Ependymoma – Ependymoma is a type of tumor that arises from ependymal cells lining the ventricles of the brain and the center of the spinal cord. It can occur in both children and adults, with varying grades of severity as classified by the World Health Organization (WHO). The tumor can present in different forms, including cellular, papillary, myxopapillary, clear-cell, tanycytic, or anaplastic variants. Ependymomas can cause symptoms by blocking the flow of cerebrospinal fluid, leading to increased pressure in the brain. The progression of the disease depends on the tumor's location, size, and grade, which can affect neurological function. Treatment often involves surgical resection, and the extent of removal can influence the disease's progression.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-512222-28-00Protocol codeET-13-002Estimated enrolment1 234 patientsSponsorCentre Leon Berard

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).