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Study on the Safety and Effectiveness of Sirolimus for Treating High-Grade Glioma in Children

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 brain tumor called high-grade glioma in children. High-grade gliomas are aggressive tumors that occur in the brain and can be challenging to treat. The study will use a medication called rapamycin, also known by its code name sirolimus, which is taken as an oral solution. Rapamycin is being investigated for its potential to help manage these tumors by targeting specific pathways in the body that may contribute to tumor growth.

The purpose of this study is to evaluate the safety and effectiveness of rapamycin in treating high-grade gliomas in children. Participants in the study will receive rapamycin and will be monitored for any side effects and how well the treatment works over time. The study will track the overall survival of participants, as well as how long they remain free from tumor progression or other events related to the disease. The study will also assess the overall response rate to the treatment, which includes measuring any changes in the tumor size or condition.

Throughout the study, researchers will carefully observe and record any adverse reactions to rapamycin, especially those that are more severe. The study aims to provide valuable information on the potential benefits and risks of using rapamycin for treating high-grade gliomas in children, contributing to the understanding and management of this challenging condition.

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

    Introduction to the study

    Upon joining the study, the patient will receive an overview of the trial's purpose, which is to evaluate the safety and effectiveness of rapamycin in treating high-grade glioma in children.

    The patient will be informed about the study's primary objective, which is to determine the safety and tolerability of rapamycin.

  2. Step 2

    Medication administration

    The patient will receive Rapamune 1 mg/mL oral solution, which contains the active substance sirolimus.

    The medication will be administered orally. The specific dosage and frequency will be determined by the study protocol and the patient's individual needs.

  3. Step 3

    Treatment phase

    During the treatment phase, the patient will be monitored for any adverse reactions. The primary safety endpoint is the assessment of the incidence of grade 3 and 4 adverse reactions.

    The patient will be evaluated for overall survival (OS), event-free survival (EFS), and progression-free survival (PFS) at various intervals: 15 months, 24 months, 3 years, and 5 years.

  4. Step 4

    Follow-up

    After the treatment phase, the patient will continue to be monitored for any long-term effects and overall health outcomes.

    The study will assess the overall response rate (ORR) and evaluate the seriousness of any adverse events according to established criteria.

Who can join the trial?

5 criteria

  • Age between 3 and 18 years old.
  • Parents or legal guardians must sign an informed consent form for children under 13. Patients over 13 must also sign the consent form. This form allows participation in study procedures, including special tests that look at genes to help understand the disease better.
  • A confirmed diagnosis of **glioma** of **WHO grade III or IV**. Glioma is a type of brain tumor, and WHO grades III and IV indicate more serious forms.
  • If the patient is of reproductive age, a pregnancy test must be done within two weeks before starting the study, and it must show that the patient is not pregnant.
  • Sexually active patients must agree in writing to use effective birth control methods.

Who cannot join the trial?

9 criteria

  • Patients who are not diagnosed with high grade glioma. This is a type of brain tumor.
  • Patients who are not within the specified age range for the study. The study is for children.
  • Patients who have any other medical conditions that might interfere with the study.
  • Patients who are unable to follow the study procedures or instructions.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent surgery or medical procedure that might affect the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have allergies or reactions to the study medication.
  • Patients who have a history of certain medical conditions that could affect the study results.
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Investigated drugs

Rapamycin is a medication being studied for its safety and effectiveness in treating high-grade gliomas, which are a type of brain tumor, in children. This trial is part of a larger effort to manage rare and very rare diseases of the central nervous system that are linked to the activation of the mTOR pathway. The main goal is to see how well children tolerate this medication and to ensure it is safe for use in this context.

What is already known about the treatment

Rapamycin – Rapamycin is administered orally in the form of tablets or capsules. It is currently being studied in clinical trials for its safety and effectiveness in treating high-grade gliomas, particularly in children. The main therapeutic use being explored is for brain tumors associated with the mTOR pathway. At the molecular level, rapamycin works by inhibiting the mTOR protein, which plays a role in cell growth and proliferation. It is classified as an immunosuppressant and is also known for its use in preventing organ transplant rejection.

Investigated diseases

High-Grade Glioma – High-grade glioma is a type of brain tumor that originates from glial cells, which support and protect nerve cells in the brain. It is characterized by rapid growth and aggressive behavior, often infiltrating surrounding brain tissue. Symptoms can vary depending on the tumor's location but may include headaches, seizures, and neurological deficits such as weakness or speech difficulties. As the disease progresses, these symptoms may worsen, and additional neurological impairments can develop. High-grade gliomas are more common in adults but can also occur in children, where they may present differently. The progression of the disease is typically marked by increasing tumor size and worsening of symptoms over time.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-515951-39-00Protocol codeBraimTOR-ONKOEstimated enrolment50 patientsSponsorInstytut Pomnik Centrum Zdrowia Dziecka

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