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Study on the Safety and Effectiveness of Methotrexate and Drug Combination for Children and Young Adults with High-Risk Metastatic Medulloblastoma

Registered 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 the safety and effectiveness of chemotherapy combined with radiation therapy for patients with metastatic medulloblastoma and other similar types of brain tumors known as embryonal tumors. These are aggressive tumors that can spread to other parts of the brain and spinal cord. The study involves several chemotherapy medications, including Methotrexate, Carboplatin, Lomustine, Cyclophosphamide, Etoposide, Thiotepa, and Vinorelbine. These medications are used to kill cancer cells or stop them from growing and are administered either through an infusion into a vein or taken orally as a capsule.

The purpose of the study is to evaluate the safety of using both standard and high doses of these chemotherapy drugs along with radiation therapy that targets the brain and spinal cord. The study is designed for children and young adults who have been newly diagnosed with high-risk medulloblastoma or other embryonal tumors. Participants will receive treatment over a period of time, and their health will be closely monitored to assess any side effects and the overall effectiveness of the treatment.

Throughout the study, researchers will track the participants' progress, including how long they live without the disease getting worse, known as progression-free survival, and their overall survival. The study will also look at any changes in neurological and psychological health, as well as any other health issues that may arise as a result of the treatment. The goal is to find the best way to treat these challenging types of brain tumors while ensuring the safety and well-being of the participants.

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 reviewing medical history and performing necessary tests to ensure the patient meets the criteria for participation.

  2. Step 2

    Treatment phase 1: standard chemotherapy

    The first phase involves administering standard chemotherapy drugs. These include methotrexate, carboplatin, lomustine, cyclophosphamide, etoposide, thiotepa, and vinorelbine. Most of these medications are given through an intravenous (IV) line, except for lomustine, which is taken orally.

    The frequency and dosage of each medication are determined by the study protocol and are tailored to the patient's specific needs.

  3. Step 3

    Treatment phase 2: high-dose chemotherapy

    Following the standard chemotherapy, the patient receives high-dose chemotherapy. This phase is designed to intensify the treatment and target the cancer cells more aggressively.

    The same medications may be used at higher doses, and the administration method remains primarily intravenous.

  4. Step 4

    Craniospinal irradiation

    After completing chemotherapy, the patient undergoes craniospinal irradiation. This is a type of radiation therapy that targets the brain and spinal cord to eliminate any remaining cancer cells.

    The duration and frequency of the irradiation sessions are specified by the treatment plan.

  5. Step 5

    Follow-up and monitoring

    Regular follow-up appointments are scheduled to monitor the patient's response to treatment and manage any side effects. This includes physical examinations, imaging tests, and laboratory tests.

    The patient's progress is evaluated based on criteria such as progression-free survival and overall survival.

  6. Step 6

    End of study

    The study is estimated to conclude by December 31, 2025. At this point, a final assessment is conducted to evaluate the overall outcomes and any long-term effects of the treatment.

Who can join the trial?

13 criteria

  • Patients must have a first-time diagnosis of high-risk **medulloblastoma** or other **embryonal tumors**. These are specific types of brain tumors.
  • The diagnosis must be according to the **WHO 2016 classification**. This is a system used to classify and describe different types of tumors.
  • The patient must not have received any previous treatment like chemotherapy or radiotherapy, except for surgery.
  • Written informed consent must be obtained from the patient, their parents, or a legal representative. This means they agree to participate in the study after understanding all the details.
  • The patient must be available for treatment and able to follow the study's rules and procedures.
  • Patients with specific types of embryonal tumors, such as **CNS neuroblastoma** or **CNS ganglioneuroblastoma**, are eligible if they meet the criteria.
  • Patients with a first-time diagnosis of **Pinealoblastomas** are eligible if they have only been treated surgically.
  • Both males and females aged over 3 years and under 21 years at the time of diagnosis can participate.
  • The patient must have a life expectancy of 12 months or more.
  • The patient must have a **Karnofsky/Lansky score** of 40% or higher. This score measures the patient's ability to carry out daily activities.
  • The patient must have adequate **hematological function**, meaning their blood cell levels are within a certain range.
  • The patient must have adequate **liver function**, meaning their liver is working properly.
  • The patient must have adequate **renal function**, meaning their kidneys are working properly.

Who cannot join the trial?

4 criteria

  • Patients who have a different type of cancer than the ones being studied, such as **metastatic medulloblastoma** or other **embryonal tumors**. These are specific types of brain tumors.
  • Patients who are not within the age range specified for the study. The study is for children and young adults.
  • Patients who are not able to undergo the treatments being tested, which include **chemotherapy** (a treatment using drugs to kill cancer cells) and **craniospinal irradiation** (a type of radiation therapy that targets the brain and spine).
  • Patients who are not considered part of the vulnerable population selected for the study. This means the study is focused on a specific group of patients who may need special consideration.
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Investigated drugs

  • Standard Chemotherapy

    is a treatment that uses drugs to kill cancer cells. In this trial, it is used as a first-line treatment for patients with high-risk medulloblastoma and other embryonal tumors. The goal is to reduce the size of the tumors and stop them from growing.

  • High-Dose Chemotherapy

    involves giving higher amounts of chemotherapy drugs to more aggressively target and destroy cancer cells. This approach is used in patients who may not respond as well to standard doses, aiming to improve the chances of eliminating the cancer.

  • Craniospinal Irradiation

    is a type of radiation therapy that targets the brain and spinal cord. It is used in this trial to treat metastatic medulloblastoma and other embryonal tumors, helping to control the spread of cancer cells throughout the central nervous system.

What is already known about the treatment

Standard and High-Dose Chemotherapy – This treatment is administered intravenously, meaning it is given directly into a vein. It is currently being studied in clinical trials to assess its safety and effectiveness for treating metastatic medulloblastoma and other embryonal tumors, particularly in pediatric and young adult patients. The main therapeutic indication is for high-risk medulloblastoma and similar tumors. The chemotherapy works by targeting and killing rapidly dividing cancer cells at the molecular level, disrupting their growth and replication. It is classified as an antineoplastic agent, which is a type of medication used to treat cancer.

Investigated diseases

  • Metastatic Medulloblastoma

    This is a type of brain cancer that originates in the cerebellum, the part of the brain that controls balance and coordination. It is considered metastatic when it spreads from its original site to other parts of the brain or spinal cord. The disease often progresses rapidly, affecting neurological functions and potentially leading to symptoms such as headaches, nausea, and balance issues. As it advances, it may cause increased intracranial pressure and other neurological complications.

  • Embryonal Tumors

    These are a group of rare, fast-growing tumors that typically occur in the brain and spinal cord. They originate from embryonic cells, which are cells that remain from the earliest stages of development. These tumors can spread to other parts of the central nervous system and may cause a variety of symptoms depending on their location, such as headaches, vomiting, and changes in behavior or personality. As they progress, they can lead to significant neurological impairment.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-518964-11-01Protocol codeMBMET_MEYER2017Estimated enrolment18 patientsSponsorAzienda Ospedaliera Universitaria Meyer IRCCS

sourced from the EU Clinical Trials Register and site verification

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