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Study on the Effectiveness of Sirolimus and Trametinib for Treating Diffuse Intrinsic Pontine Glioma in Children Aged 3-18 Years

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 a rare and aggressive brain tumor called Diffuse Intrinsic Pontine Glioma (DIPG), which primarily affects children. The study aims to find the best treatment for children aged 3 to 18 years by using drugs that target specific molecular markers found in the tumor. The medications being tested in this study are Sirolimus and Trametinib. Sirolimus is available as an oral solution and coated tablets, while Trametinib is provided as film-coated tablets. These drugs are chosen based on the unique molecular profile of each child's tumor.

The purpose of the study is to develop an optimal treatment plan for children with DIPG by identifying important molecular markers and adjusting the type of medication accordingly. The study will assess the safety and effectiveness of Sirolimus and Trametinib in treating DIPG. Participants will receive these medications either alone or in combination, depending on their specific tumor characteristics. The study will monitor the overall survival of participants from the time of diagnosis and evaluate the safety of Sirolimus when used during radiotherapy and in combination with Trametinib after irradiation.

Throughout the study, the progression of the disease will be closely monitored, and the response to treatment will be assessed using MRI scans every two months. Additionally, the quality of life of participants will be evaluated using a pediatric quality of life inventory. The study will also compare the safety of treatment with Sirolimus alone versus in combination with Trametinib. The estimated end date for the study is June 2026.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    The study begins with an initial assessment to confirm eligibility. This includes verifying age between 3 and 18 years and a diagnosis of diffuse intrinsic pontine glioma (DIPG) based on clinical and radiological criteria.

    A histological diagnosis of glioma WHO grade III and IV must be confirmed by a reference pathologist.

  2. Step 2

    Consent process

    Informed consent must be signed by the parents or legal guardian of the minor. If the patient is over 13 years of age, they must also provide consent.

    The consent covers study diagnostic and therapeutic procedures, including molecular-based diagnostic and prognostic tests using the NGS method.

  3. Step 3

    Molecular profiling

    Molecular profiling is conducted to identify important markers for therapy. This helps in adjusting the type of medicinal product to the indicated markers.

  4. Step 4

    Treatment initiation

    Treatment begins with the administration of sirolimus during radiotherapy. Sirolimus is available as a 1 mg/mL oral solution or 1 mg coated tablets, taken orally.

    After radiotherapy, trametinib is introduced as an adjunctive treatment. Trametinib is available as 0.5 mg film-coated tablets, also taken orally.

  5. Step 5

    Treatment monitoring

    Regular monitoring is conducted to assess the safety and efficacy of the treatment. This includes MRI scans every 2 months to evaluate response rate and progression-free survival.

    Quality of life is assessed using the PedQoL (pediatric quality of life inventory) at specified time points.

  6. Step 6

    Safety and efficacy evaluation

    The primary endpoint is overall survival from the time of diagnosis until the last visit or death.

    Safety is assessed for sirolimus administered during radiotherapy and in combination with trametinib after irradiation.

  7. Step 7

    Study completion

    The study is estimated to end by June 30, 2026. The final evaluation will include a comparison of safety in two groups: those treated with sirolimus alone and those treated with sirolimus in combination with trametinib.

Who can join the trial?

4 criteria

  • Age between 3 and 18 years old.
  • Parents or legal guardians must sign an informed consent form. If the patient is over 13 years old, they must also sign the form. This form gives permission for the study's tests and treatments, including special tests that look at genes using a method called NGS (Next-Generation Sequencing).
  • The patient must have a diagnosis of diffuse intrinsic pontine glioma (DIPG), which is determined by looking at symptoms and imaging tests like MRI.
  • A tissue sample must show a diagnosis of glioma that is classified as WHO grade III or IV. This must be confirmed by a specialist pathologist. A pathologist is a doctor who examines tissues to diagnose diseases.

Who cannot join the trial?

4 criteria

  • Patients who do not have a diagnosis of diffuse intrinsic pontine glioma (DIPG) cannot participate. DIPG is a type of brain tumor that occurs in a specific area of the brainstem.
  • Patients who are not within the specified age range for the study cannot participate. The study is for children, so adults are excluded.
  • Patients who are not able to safely undergo the treatments being tested in the study cannot participate. This is to ensure the safety of all participants.
  • Patients who are unable to comply with the study procedures or follow-up requirements cannot participate. This is important for the accuracy and reliability of the study results.
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Investigated drugs

  • Sirolimus

    is a medication that helps to suppress the immune system. In this study, it is being used to see if it can help treat children with a type of brain tumor called DIPG by targeting specific molecular markers identified in the tumor.

  • Trametinib

    is a medication that works by blocking certain proteins that help cancer cells grow. In this trial, it is being tested to determine if it can effectively treat DIPG in children by focusing on specific molecular markers found in the tumor.

What is already known about the treatment

  • Sirolimus

    Sirolimus is administered orally and is currently being studied for its potential use in treating diffuse intrinsic pontine glioma (DIPG) in children. It is an immunosuppressant that is well-documented in medical literature for its use in preventing organ transplant rejection. The main therapeutic indication in this trial is to target specific molecular markers associated with DIPG. Sirolimus works by inhibiting a protein called mTOR, which plays a role in cell growth and proliferation. It is classified as an mTOR inhibitor.

  • Trametinib

    Trametinib is taken orally and is under investigation for its effectiveness in treating diffuse intrinsic pontine glioma (DIPG) in children. It is recognized in medical literature for its use in treating certain types of cancer, such as melanoma. The primary therapeutic indication in this study is to address molecular markers linked to DIPG. Trametinib functions by inhibiting MEK1 and MEK2 proteins, which are part of a pathway that helps control cell division and survival. It is classified as a MEK inhibitor.

Investigated diseases

Diffuse Intrinsic Pontine Glioma – This is a rare and aggressive brain tumor that primarily affects children. It occurs in the pons, a part of the brainstem, which controls essential functions such as breathing and heart rate. The tumor grows rapidly and infiltrates the surrounding brain tissue, making it difficult to treat surgically. As it progresses, it can cause symptoms such as difficulty with balance and coordination, problems with eye movements, and facial weakness. Over time, these symptoms may worsen, affecting the child's ability to walk, speak, and swallow. The disease is challenging to manage due to its location and aggressive nature.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515952-19-00Protocol codeDIPGenEstimated enrolment100 patientsSponsorInstytut Pomnik Centrum Zdrowia Dziecka

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