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Study on Trametinib Dimethyl Sulfoxide for Children with Refractory Histiocytosis Not Responding to Conventional Treatment

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 a condition known as histiocytic cell proliferation, which involves an abnormal increase in certain immune cells. The trial is testing a medication called Trametinib Dimethyl Sulfoxide, which is taken as a film-coated tablet. The purpose of the study is to evaluate the safety, effectiveness, and how well patients tolerate this drug, as well as to determine the best duration of treatment for children whose condition has not improved with standard therapies.

Participants in the study will receive the medication Trametinib to see if it can help manage their condition, especially if they have not responded to other treatments like Vemurafenib. The study will also include a comparison with a placebo to better understand the drug's effects. The trial will monitor participants over a period to assess their response to the treatment and any side effects they may experience.

The study aims to find the right dose of Trametinib for children under 18, ensuring it is similar to the dose recommended for adults. Throughout the trial, various health checks, including laboratory tests and heart monitoring, will be conducted to ensure the safety of the participants. The trial will also look at how long patients can live without the disease getting worse and the overall survival rate. The study is expected to continue until March 2026.

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

    Joining the study

    Participation begins after signing an informed consent form. This document confirms understanding and agreement to participate in the study, including the use of the drug trametinib.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to confirm eligibility. This includes checking for the absence of mutations in the BRAF gene or previous failure of vemurafenib treatment.

    The assessment may involve reviewing medical history and previous treatments, such as vinblastine and prednisolone, or other second-line treatments.

  3. Step 3

    Treatment with trametinib

    The drug trametinib dimethyl sulfoxide is administered orally. The dosage and duration are optimized based on individual needs and responses.

    Regular monitoring is conducted to assess the safety and effectiveness of the treatment. This includes checking vital signs, laboratory tests, and performing echocardiography and ECG.

  4. Step 4

    Monitoring and follow-up

    Throughout the treatment, event-free survival (EFS) is monitored, along with any adverse events.

    Progression-free survival (PFS), overall survival (OS), and overall response rate (ORR) are also evaluated.

    The reactivation rate of the disease is assessed after two years.

  5. Step 5

    Completion of the study

    The study is estimated to conclude by March 30, 2026. Upon completion, a final assessment is conducted to evaluate the overall outcomes and any long-term effects of the treatment.

Who can join the trial?

11 criteria

  • The patient must have a condition called **histiocytic cell proliferation**.
  • The patient must be a child or teenager, as the study is for **juvenile patients**.
  • The patient must not have changes, called **mutations**, in a specific part of their genes known as the **BRAF gene**. If they do have these changes, they must have tried a treatment called **Vemurafenib** and it did not work.
  • The patient must have tried other treatments and they did not work. This includes:
    • The disease got worse during the first or second treatment, which should have included a medicine called **Vinblastine** and another called **prednisolone**, or other medicines like **Cytosine Arabinoside** or **Cladribine**.
    • The disease came back after getting better with the first treatment.
    • The disease got worse during or after using **Vemurafenib**.
    • The patient shows signs of a brain condition seen in an **MRI** scan.
    • The patient or their guardian must agree to participate in the study by signing a form called **informed consent**.
    • If the patient is old enough to have children, they must agree to use **effective contraception** during the study and for at least one year after stopping the study medicine.
    • The patient must be part of another study called the **HISTIOGEN trial**.
    • The study is open to both **male and female** patients.

Who cannot join the trial?

8 criteria

  • Patients who have a different condition than **histiocytic cell proliferation**. This is a condition where certain immune cells grow too much.
  • Patients who are not **juvenile**. This means the study is only for children and young people.
  • Patients who have not tried **Vemurafenib** treatment before. Vemurafenib is a medication used to treat certain types of cancer.
  • Patients who are not **BRAF negative**. BRAF is a gene, and being BRAF negative means the gene does not have a specific mutation.
  • Patients who are not **refractory** to treatment. Refractory means the disease does not respond to treatment.
  • Patients who are not within the specified **age range**. The study is for a specific age group.
  • Patients who are not part of the specified **clinical trial group**. This means the study is for a specific group of patients.
  • Patients who are not considered part of a **vulnerable population**. This refers to groups who may need special protection in research.
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Investigated drugs

  • Trametinib

    is a medication used in this trial to help treat juvenile patients with a condition called refractory histiocytosis. This condition is difficult to treat, and Trametinib is being tested to see if it can help improve the symptoms or manage the disease better. The trial aims to find the best time and amount of Trametinib to use for these patients.

  • Vemurafenib

    is another medication that some patients in the trial may have used before. It is typically used to treat certain types of cancer, but in this trial, it is relevant because the patients have not responded well to it. The trial is exploring if switching to Trametinib can be more effective for these patients.

What is already known about the treatment

Trametinib – Trametinib is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in treating histiocytic cell proliferation, particularly in juvenile patients who are BRAF negative and have refractory histiocytosis or have not responded to Vemurafenib treatment. The medication works by inhibiting a specific protein in the cell signaling pathway, which helps to slow down or stop the growth of abnormal cells. Trametinib is classified as a MEK inhibitor, a type of targeted cancer therapy.

Investigated diseases

Histiocytic Cell Proliferation – This condition involves an abnormal increase in the number of histiocytes, which are a type of immune cell. These cells can accumulate in various tissues and organs, potentially leading to damage and dysfunction. The proliferation can cause symptoms depending on where the cells gather, such as skin rashes, bone pain, or organ enlargement. It may progress slowly or rapidly, affecting the patient's quality of life. The condition is considered rare and can vary significantly in its presentation and severity. Understanding the underlying cause of the proliferation is crucial for managing the disease effectively.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-515896-37-00Protocol codeTRAMEstimated enrolment12 patientsSponsorInstytut Matki I Dziecka

sourced from the EU Clinical Trials Register and site verification

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