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Study on Vemurafenib for Children with BRAF Mutation-Resistant Histiocytosis

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 specifically looking at cases where this condition is resistant to standard treatments. The study will use a medication called vemurafenib, which is taken as a film-coated tablet. Vemurafenib is designed to target a specific genetic change, known as the BRAF mutation, that can occur in these cells.

The purpose of the study is to evaluate the safety, effectiveness, and tolerability of vemurafenib in children who have this BRAF mutation and whose condition has not responded to other treatments. Participants will receive vemurafenib and will be monitored to determine the best dosage and duration of treatment. The study will also assess how well the drug works in preventing the disease from getting worse and how long patients can live without the disease progressing.

Throughout the study, various health checks will be conducted, including monitoring vital signs, performing laboratory tests, and using imaging techniques like MRI to ensure the safety of the participants. The trial aims to find a dose for children that provides similar benefits as those seen in adults. The study will also look at how often the disease comes back after two years of treatment. Participants will be closely observed for any side effects or adverse events during the trial.

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

    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 vemurafenib.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to confirm the presence of mutations in the BRAF gene in tumor tissues or circulating tumor DNA (ctDNA).

    Eligibility is confirmed based on previous treatment history and current health status.

  3. Step 3

    Treatment phase

    The treatment involves taking vemurafenib orally. The dosage and frequency are determined based on individual needs to achieve drug exposure similar to that recommended for adults.

    The duration of treatment is optimized for each patient, focusing on safety and effectiveness.

  4. Step 4

    Monitoring and evaluation

    Regular monitoring includes checking vital signs, laboratory tests, echocardiography, and electrocardiogram (ECG) findings.

    Adverse events are assessed to ensure the safety of vemurafenib treatment.

  5. Step 5

    End of treatment

    The treatment phase concludes based on the achievement of specific health outcomes or the occurrence of certain events.

    Event-free survival (EFS) and other health metrics such as progression-free survival (PFS) and overall survival (OS) are evaluated.

  6. Step 6

    Follow-up

    After treatment, follow-up assessments are conducted to monitor long-term health outcomes, including the reactivation rate of the disease after two years.

    Patients are advised to use effective contraception during the treatment period and for at least one year after discontinuation if applicable.

Who can join the trial?

11 criteria

  • The patient must have a specific change, called a mutation, in the BRAF gene found in their tumor or in their blood at any time during treatment or follow-up.
  • The patient must have experienced treatment failure, which means the treatment did not work. This can be shown by one of the following:
    • The disease got worse during the first or second treatment, affecting at least one important organ. The patient should have had at least 6 weeks of a drug called Vinblastine with at least 28 days of prednisolone, or at least 2 cycles of another drug called Cytosine Arabinoside or Cladribine as a second treatment.
    • The disease came back after initially getting better with Vinblastine and prednisolone, or did not respond to the second treatment with Cytosine Arabinoside or Cladribine, or affected at least one important organ.
    • The disease came back for the third time or more, with or without affecting important organs.
    • The disease came back after finishing treatment with a drug called Vemurafenib.
    • Signs of a neurodegenerative disorder (a condition affecting the brain) appeared in an MRI (a type of scan) of the CNS (central nervous system).
    • The patient or their guardian must sign a form agreeing to participate in the trial and receive Vemurafenib treatment, following legal rules.
    • The patient must agree to use effective birth control during the time they are taking Vemurafenib and for at least 1 year after stopping the treatment, if they are at an age where they can have children.
    • The patient must be part of the HISTIOGEN trial.
    • The trial is open to both male and female patients.
    • The trial includes patients who are considered a vulnerable population, which means they may need extra protection or care.

Who cannot join the trial?

4 criteria

  • Patients who do not have a condition called histiocytic cell proliferation cannot participate. This condition involves an increase in certain types of cells in the body.
  • Patients who are not BRAF positive cannot participate. This means they do not have a specific genetic change in their cells.
  • Patients who are not juvenile cannot participate. This means the study is only for children and young people.
  • Patients who do not have refractory histiocytosis cannot participate. This means their condition does not respond to standard treatments.
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Investigated drugs

Vemurafenib is a medication used in this clinical trial to treat juvenile patients who have a specific type of histiocytosis that is difficult to manage with other treatments. This condition is characterized by the presence of a mutation in the BRAF gene. Vemurafenib works by targeting and inhibiting the activity of the mutated BRAF protein, which is involved in the growth and spread of cancer cells. The trial aims to find the best timing and amount of Vemurafenib to use for these young patients to improve their treatment outcomes.

What is already known about the treatment

Vemurafenib – Vemurafenib is administered orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness in treating BRAF-positive juvenile patients with refractory histiocytosis, a condition characterized by excessive growth of histiocytic cells. The medication works by inhibiting the BRAF protein, which is involved in cell growth and division, thereby slowing down or stopping the proliferation of these cells. Vemurafenib is classified as a BRAF 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 the affected areas, such as skin rashes, bone pain, or organ enlargement. It is often associated with genetic mutations, such as the BRAF mutation, which can influence the behavior of these cells. The progression of the disease can vary, with some cases remaining stable while others may worsen over time. Understanding the specific genetic and molecular characteristics of the condition is crucial for managing its progression.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-515805-25-00Protocol codeBRAVOEstimated enrolment25 patientsSponsorInstytut Matki I Dziecka

sourced from the EU Clinical Trials Register and site verification

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