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Comparing two treatment regimens with drug combination for newly diagnosed low-risk medulloblastoma to evaluate cognitive function outcomes

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 focuses on studying medulloblastoma, specifically the non-metastatic, SHH-activated type in young patients. Medulloblastoma is a type of brain tumor that typically develops in children. The study aims to compare brain function outcomes between two different treatment approaches.

The treatment involves several chemotherapy medications including methotrexate, etoposide, cyclophosphamide, carboplatin, vincristine, cisplatin, and thiotepa. These medications will be given through different methods, including directly into the bloodstream through a vein and, in some cases, directly into areas around the brain.

The study will track how well children's thinking and learning abilities develop over time after receiving treatment. Doctors will measure these abilities through various tests when the treatment starts and continue monitoring for several years afterward. They will also check for any hearing problems and changes in brain tissue that might occur as a result of the treatment.

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

    Initial assessment

    Your diagnosis will be confirmed as medulloblastoma (a type of brain tumor) that meets specific medical criteria

    You will be randomly assigned to one of two treatment groups: Group A ('Head Start' 4) or Group B (HITSKK)

  2. Step 2

    Treatment administration

    You will receive medications through intravenous (into the vein) administration

    The medications include: methotrexate (given both intravenously and through the brain fluid), etoposide, cyclophosphamide, carboplatin, vincristine, cisplatin, and thiotepa

  3. Step 3

    Initial evaluation

    Your mental abilities will be assessed using age-appropriate tests

    Quality of life questionnaires will be completed

    Hearing tests will be performed

  4. Step 4

    2.5-year follow-up

    Mental ability tests will be repeated

    Quality of life assessment will be conducted

    Hearing evaluation will be performed

    Brain imaging will be done to check for any white matter changes

  5. Step 5

    5-year follow-up

    Final set of mental ability tests will be conducted

    Quality of life will be assessed again

    Final hearing evaluation will be performed

    Brain imaging will be repeated to check for any changes

Who can join the trial?

7 criteria

  • Child must be under 5 years old at the time of diagnosis
  • Child must have suspected or confirmed SHH-activated medulloblastoma (a specific type of brain tumor) as determined by the medical facility
  • Family must be able to commit to regular follow-up appointments for brain function testing
  • Parents or legal guardians must be able to understand the study information and provide written consent for screening procedures
  • Both the child and parents/legal guardians must be willing and able to participate in the entire duration of the study if the child qualifies
  • Both male and female children can participate in the study
  • Family must have stable living circumstances that allow for consistent participation in the study

Who cannot join the trial?

12 criteria

  • Prior treatment with chemotherapy, radiotherapy or other anti-cancer therapy (except for surgery)
  • Presence of metastatic disease (cancer that has spread to other parts of the body)
  • Having a TP53 mutation (a specific genetic change in tumor cells)
  • Having MYC gene amplification (increased copies of a specific cancer-related gene)
  • Presence of other types of brain tumors not classified as SHH-activated medulloblastoma
  • Severe medical conditions that could interfere with study treatment
  • Inability to undergo required medical procedures or tests
  • Active infections that require treatment
  • Pregnancy or breastfeeding
  • Participation in other clinical trials within 30 days before starting this study
  • Known allergies or hypersensitivity to study medications
  • Mental conditions that could affect the ability to follow study requirements
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Investigated drugs

  • Carboplatin

    is a chemotherapy drug given through a vein (intravenously). In this trial it is used to help stop the growth of cancer cells in the brain by damaging their DNA, which can improve the chances of controlling the tumor.

  • Cyclophosphamide

    is an intravenous chemotherapy medication. It works by interfering with the ability of cancer cells to divide and grow, helping to shrink the tumor and prevent it from spreading.

  • Etoposide

    is given by IV infusion. It attacks cancer cells by preventing them from copying their DNA, which slows down or stops tumor growth.

  • Thiotepa

    is an IV chemotherapy agent. It damages the DNA of cancer cells, making it harder for the tumor to grow and spread.

  • Methotrexate

    (given intravenously) is a chemotherapy drug that blocks a substance cancer cells need to make DNA. This stops the cells from multiplying and helps reduce the tumor size.

  • Methotrexate

    (given directly into the spinal fluid, intraventricular use) is used to reach cancer cells that may be in the brain’s fluid spaces. By delivering the drug right where the tumor is, it can more effectively kill any remaining cancer cells.

What is already known about the treatment

  • CARBOPLATIN

    Given by slow injection into a vein, carboplatin is an approved cancer drug that has been used for many years. It works by attaching to DNA in cancer cells, which stops the cells from copying their genetic material and leads to their death. It belongs to the class of platinum‑based chemotherapy agents and is mainly used to treat cancers such as ovarian, lung, and brain tumors. In medical literature it is well‑studied and considered a standard part of many treatment regimens.

  • CYCLOPHOSPHAMIDE

    This medication is delivered intravenously as a drip and is an established chemotherapy drug. Inside the body it is changed into an active form that damages DNA, preventing cancer cells from growing and causing them to die. It is classified as an alkylating agent and is commonly used for lymphomas, breast cancer, and certain solid tumors. It has a long history of use and is fully described in the medical literature.

  • ETOPOSIDE

    Administered by IV infusion, etoposide is a widely accepted cancer medicine. It stops cancer cells from repairing their DNA by blocking an enzyme needed for DNA unwinding, which leads to cell death. This drug is part of the topoisomerase‑II inhibitor class and is often used for lung cancer, testicular cancer, and some brain tumors. It is a well‑known, approved therapy with extensive research support.

  • THIOTEPA

    Given through an IV injection, thiotepa is a long‑standing chemotherapy agent. It works by adding alkyl groups to DNA, which breaks the DNA strands and kills rapidly dividing cancer cells. It is categorized as an alkylating agent and is used for cancers such as brain tumors, ovarian cancer, and certain blood cancers. Its safety and effectiveness are documented in many clinical studies.

  • METHOTREXATE

    Methotrexate can be given either by IV infusion or directly into the spinal fluid (intrathecal) for this trial; both methods involve injecting the drug into the bloodstream or spinal canal. It blocks an enzyme needed for making DNA building blocks, so cancer cells cannot grow and eventually die. This drug is a folate antagonist and is used for many cancers, including leukemia, lymphoma, and brain tumors, as well as for some non‑cancer diseases. It is an approved, well‑researched medication with decades of clinical use.

  • VINCRISTINE

    Delivered by slow IV infusion, vincristine is a standard chemotherapy drug. It stops cancer cells from dividing by attaching to tiny tubes inside the cell that pull chromosomes apart, causing the cells to stop growing and die. It belongs to the class of vinca alkaloids and is commonly used for leukemias, lymphomas, and solid tumors such as neuroblastoma. Its role in cancer treatment is well established in medical guidelines.

Investigated diseases

  • Medulloblastoma

    A brain tumor that starts in the cerebellum, the part of the brain that controls balance and coordination. It develops from primitive or embryonic cells that remain in the brain after birth. The tumor typically forms in the lower back part of the brain and can spread to other parts of the brain and spinal cord through the cerebrospinal fluid. Medulloblastoma grows relatively quickly and can interfere with normal brain function, affecting movement, balance, and other basic functions.

  • Gorlin Syndrome

    A genetic condition that affects multiple systems in the body, also known as Basal Cell Nevus Syndrome. People with this condition are born with abnormalities in multiple body parts and tend to develop various types of tumors. The syndrome causes distinctive facial features, multiple small skin growths, and skeletal abnormalities. It can also lead to the development of jaw cysts and calcification in various parts of the brain.

  • Leukoencephalopathy

    A condition that affects the white matter of the brain, characterized by deterioration of the brain's white matter. The white matter contains nerve fibers that are covered by myelin, which helps transmit signals in the brain. This condition can develop gradually and affects how different parts of the brain communicate with each other. It can impact various brain functions including movement and thinking abilities.

Trial detailsLast updated 21 Oct 2026
Age0-17PhasePhase IIITrial ID2024-517133-40-00Protocol codeCOGNITO-MBEstimated enrolment46 patientsSponsorGPOH gGmbH

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