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Study on Selumetinib for Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

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 study focuses on a condition known as Neurofibromatosis Type 1 (NF1), which can cause non-cancerous tumors called plexiform neurofibromas. These tumors are symptomatic and cannot be operated on, making them a challenge to treat. The investigation will explore the effectiveness and safety of a drug named Selumetinib, taken in capsule form, compared to a placebo. The goal of the research is to evaluate how well Selumetinib works in treating these tumors.

Participants in the study will receive either Selumetinib or a placebo without knowing which one they are taking to ensure impartiality in the results. The focus will be on monitoring changes in the size and symptoms of the tumors and other related health aspects. Researchers will use a type of imaging called volumetric MRI to assess the effectiveness of the treatment by measuring any changes in tumor size.

Through this approach, the study aims to determine whether Selumetinib can help manage NF1-related plexiform neurofibromas by reducing tumor size and alleviating symptoms. The results will help understand if Selumetinib is a viable treatment for this 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

    Enrollment

    Upon joining the study, eligibility is confirmed. This includes being an adult aged 18 or older with a diagnosis of Neurofibromatosis Type 1 (NF1) and having symptomatic, inoperable plexiform neurofibromas (PN).

    A target PN must be measurable by MRI, and chronic PN pain must be documented. Stable use of pain medication is required, along with adequate organ and marrow function.

  2. Step 2

    Randomization

    Participants are randomly assigned to one of two groups: one receiving selumetinib and the other receiving a placebo. This process is double-blind, meaning neither the participant nor the researchers know which treatment is being administered.

  3. Step 3

    Treatment administration

    Participants in the selumetinib group take the medication orally. The dosage is either 10 mg or 25 mg, depending on the specific requirements of the study.

    The placebo group receives capsules that match the selumetinib in appearance but contain no active medication.

  4. Step 4

    Monitoring and assessments

    Regular monitoring occurs throughout the study. This includes MRI scans to assess changes in the size of the PN and evaluations of pain intensity and quality of life.

    Participants' responses to the treatment are measured using specific criteria, including the Objective Response Rate (ORR) and other health-related quality of life assessments.

  5. Step 5

    Completion

    The study is estimated to conclude by May 25, 2025. At the end of the trial, data from all participants are analyzed to determine the efficacy and safety of selumetinib compared to the placebo.

Who can join the trial?

7 criteria

  • Must be an adult aged 18 years or older.
  • Must have a diagnosis of Neurofibromatosis Type 1 (NF1), which is a genetic disorder that causes tumors to grow on nerves.
  • Must have symptomatic, inoperable Plexiform Neurofibromas (PN), meaning the tumors cause symptoms and cannot be removed by surgery.
  • Must have at least one target PN that can be measured using a special type of scan called volumetric MRI.
  • Must have a record of chronic pain from the target PN for a certain period during the screening phase.
  • Must be using stable medication for chronic PN pain at the time of joining the study.
  • Must have adequate function of organs and bone marrow, which are important for overall health and blood cell production.

Who cannot join the trial?

7 criteria

  • Patients who do not have Neurofibromatosis Type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas (PN) cannot participate. NF1 is a genetic disorder that causes tumors to grow on nerves, and PN are a type of tumor that cannot be removed by surgery.
  • Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
  • Patients who are not willing or able to follow the study procedures and requirements cannot participate.
  • Patients who have other medical conditions that might interfere with the study or its results cannot participate.
  • Patients who are taking medications that might interfere with the study treatment cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible to participate.
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Investigated drugs

Selumetinib is a medication being studied for its potential to help people with a condition called NF1, which can cause tumors to grow on nerves. These tumors, known as plexiform neurofibromas, can be painful and difficult to remove with surgery. The trial is investigating whether selumetinib can reduce the size of these tumors and improve symptoms by using MRI scans to measure changes in tumor size.

What is already known about the treatment

Selumetinib – Selumetinib is administered orally in the form of capsules. It is currently being studied in clinical trials for its effectiveness in treating Neurofibromatosis Type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas. The main therapeutic indication is to reduce the size of these tumors and alleviate symptoms. At the molecular level, selumetinib works by inhibiting the MEK1 and MEK2 proteins, which are part of a pathway that promotes cell growth and division. It is classified as a MEK inhibitor in pharmacology.

Investigated diseases

Neurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN) – This is a genetic disorder characterized by the development of multiple non-cancerous tumors along nerves in the skin, brain, and other parts of the body. These tumors, known as neurofibromas, can cause a variety of symptoms depending on their size and location. Plexiform neurofibromas are a type of tumor associated with NF1 that can grow along nerve pathways and may become large and problematic. When these tumors are symptomatic and cannot be surgically removed, they can lead to pain, disfigurement, and functional impairment. The progression of the disease can vary widely among individuals, with some experiencing mild symptoms and others facing significant challenges. The condition is considered a rare disease and is often diagnosed in childhood.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507336-20-00Protocol codeKOMET D134BC00001Estimated enrolment145 patientsSponsorAstraZeneca AB

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).