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Study on Trametinib for Patients with Neurofibromatosis Type 1 and Symptomatic Plexiform Neurofibromas

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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 called Neurofibromatosis type 1, which is a genetic disorder that often leads to the development of tumors along nerves in the skin, brain, and other parts of the body. The trial is specifically looking at a type of tumor known as plexiform neurofibromas, which can cause significant symptoms and complications. The treatment being tested in this study is a medication called Trametinib, also known by its code name GSK1120212B. Trametinib is taken in the form of film-coated tablets.

The purpose of the study is to determine if Trametinib can help reduce the size of these tumors. Participants in the study will take the medication orally, and the effects will be monitored over time. The study will use MRI scans to measure any changes in the size of the tumors. The trial will also assess other factors such as pain levels and any side effects experienced by participants.

The study is expected to continue until early 2027. Participants will be closely monitored throughout the trial to ensure their safety and to gather data on the effectiveness of Trametinib in treating plexiform neurofibromas associated with Neurofibromatosis type 1. This research aims to provide valuable insights into potential new treatments for this challenging condition.

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

    Upon joining the study, eligibility is confirmed based on specific criteria such as age, health status, and the presence of neurofibromatosis type 1 with symptomatic plexiform neurofibromas.

    A signed, written informed consent is required to participate.

  2. Step 2

    Medication administration

    The medication used in this study is trametinib, administered in the form of Mekinist 0.5 mg film-coated tablets.

    The tablets are taken orally. The exact dosage and frequency are determined by the study protocol and the treating physician.

  3. Step 3

    Treatment monitoring

    The primary goal is to observe whether trametinib can reduce the size of plexiform neurofibromas by at least 20%, as measured by volumetric MRI analysis.

    Regular monitoring includes MRI scans to assess tumor volume and other evaluations to track any changes in symptoms or side effects.

  4. Step 4

    Assessment of outcomes

    Primary outcomes focus on changes in tumor volume.

    Secondary outcomes include assessments of pain using the Numeric Pain Rating Scale (NRS-11), quality of life measures, and any adverse events.

  5. Step 5

    Completion of the study

    The study is expected to conclude by January 1, 2027.

    Final assessments will be conducted to evaluate the overall effectiveness and safety of the treatment.

Who can join the trial?

12 criteria

  • The patient must have **Neurofibromatosis type 1 (NF1)**, which is a genetic condition that causes tumors to grow on nerves.
  • The patient must have normal blood function, which means having enough red blood cells, white blood cells, and platelets.
  • The patient must have normal liver function, which means having normal levels of bilirubin and liver enzymes, unless they have a condition called Gilbert's syndrome.
  • The patient must have normal kidney function, which means having normal levels of creatinine.
  • The patient must have a **plexiform neurofibroma (PNF)**, which is a type of tumor that can cause symptoms like pain, disfigurement, or loss of function. The doctor will decide if the tumor is significant enough.
  • The patient must sign a written consent form to participate in the study.
  • The patient must be at least 18 years old.
  • The patient must have a **Karnofsky performance level** of 70% or higher, which means they can take care of themselves and do normal activities.
  • The patient must not have any standard treatment options available, meaning the tumor cannot be removed by surgery without causing serious harm.
  • The patient must have at least one measurable PNF, which is a tumor that is at least 3 cm in size in one direction.
  • The patient must be able to swallow and keep down medication taken by mouth.
  • Female patients who can have children must have a negative pregnancy test before starting the study and agree to use effective birth control.

Who cannot join the trial?

3 criteria

  • Patients who do not have Neurofibromatosis type 1, a genetic disorder that causes tumors to form on nerve tissue, cannot participate.
  • Patients who are younger than 3 years old cannot participate.
  • Patients who are part of a vulnerable population, meaning they might need special protection or care, cannot participate.
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Investigated drugs

Trametinib is a medication being studied for its ability to reduce the size of plexiform neurofibromas in patients with neurofibromatosis type 1. The goal of the trial is to see if this medication can cause the tumors to shrink by at least 20%, as measured by MRI scans.

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 neurofibromatosis type 1, specifically targeting symptomatic plexiform neurofibromas. The main therapeutic indication is to reduce the size of these tumors. At the molecular level, trametinib works by inhibiting a protein called MEK, which is part of a pathway that helps cells grow and divide. This medication is classified as a MEK inhibitor, a type of targeted cancer therapy.

Investigated diseases

Neurofibromatosis type 1 – This is a genetic disorder characterized by the development of multiple non-cancerous tumors called neurofibromas, which grow on nerves throughout the body. These tumors can vary in size and number, and they often appear on the skin as soft bumps. The condition may also cause skin changes such as café-au-lait spots, which are flat, pigmented birthmarks. Over time, individuals with this condition may experience complications such as bone deformities, learning disabilities, and vision problems. The progression of the disease can vary widely among individuals, with some experiencing mild symptoms and others facing more significant challenges. Neurofibromatosis type 1 is typically diagnosed in childhood, and its symptoms can evolve as the person ages.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-516593-30-00Protocol codeNL69517.078.19Estimated enrolment30 patientsSponsorErasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

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