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Study on Crizotinib for Treating Severe Neurofibromatosis Type 2 in Children Not Eligible for Surgery or Radiotherapy

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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 2 (NF-2), which is a genetic disorder that can lead to the growth of tumors in the nervous system. These tumors are usually non-cancerous but can cause serious health issues, especially in children. The trial is testing a medication called Crizotinib, which is taken as a capsule. The purpose of the study is to evaluate how effective Crizotinib is in treating children with severe NF-2 who cannot undergo surgery or radiotherapy.

Participants in the study will take Crizotinib orally, and the study will monitor changes in the size of the tumors using imaging techniques. The study aims to see if the tumors stabilize or shrink over time. The treatment period can last up to 60 days, and the study will continue to observe the effects for at least six months after the treatment ends.

The main goal is to determine if Crizotinib can help manage the tumors in children with NF-2, potentially improving their quality of life and extending their survival. The study is non-commercial and non-randomized, meaning it is focused on understanding the treatment's effects without comparing it to other treatments or placebos. The trial is expected to conclude by the end of 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

    Upon joining the study, eligibility is confirmed based on specific criteria, including age, ability to swallow capsules, and certain health conditions.

    The study is designed for children aged 4 to 15 with a diagnosis of neurofibromatosis type 2, a condition involving tumors in the nervous system.

  2. Step 2

    Medication administration

    The medication used in this study is crizotinib, available in two dosages: 250 mg and 200 mg hard capsules.

    The medication is taken orally, and the dosage is determined based on individual health assessments.

  3. Step 3

    Treatment duration

    The treatment involves regular administration of crizotinib, with the duration tailored to the patient's response and health status.

    The primary goal is to assess changes in tumor size through radiological evaluations.

  4. Step 4

    Monitoring and assessments

    Throughout the study, regular monitoring of health parameters is conducted to ensure safety and effectiveness.

    Assessments include imaging studies to evaluate tumor response, following specific criteria known as RECIST 1.1.

  5. Step 5

    End of treatment evaluation

    At the end of the treatment period, the primary endpoint is to achieve stabilization or regression of the primary tumor.

    Secondary endpoints include maintaining tumor stabilization or regression for at least six months post-treatment.

Who can join the trial?

14 criteria

  • Children who meet the clinical criteria for diagnosing **NF-2** (a genetic condition) with multiple tumor spots in the central or peripheral nervous system.
  • Children aged between 4 and 15 years who can swallow a tablet whole.
  • Children with:
    • **Volumetric magnetic resonance (MR) studies** showing steady tumor growth (more than 10% increase in tumor size over 4 to 12 months) to assess response using imaging criteria called **RECIST 1.1**.
    • No reasons preventing the use of the drug **crizotinib** as specified in the product information.
    • A blood system that can handle treatment, with a specific count of **neutrophils** (a type of white blood cell) and **platelets** (cells that help with blood clotting).
    • Kidney function that allows treatment, with a **creatinine level** (a waste product in the blood) not exceeding certain limits based on age.
    • **Bilirubin** (a substance made during the breakdown of red blood cells) and liver enzymes like **transaminases** and **alkaline phosphatase** within specific limits.
    • Children expected to live more than one year with a **Lansky/Karnofsky Pediatric Performance** score of 60 or higher, indicating a stable health status.
    • Children with a stable neurological condition for at least a year, without complications from previous treatments like radiotherapy or chemotherapy.
    • Children without other health conditions that could increase the risk of complications from crizotinib treatment.
    • Parents who:
      • Can care for the child, follow the treatment plan, and sign a consent form for treatment.
      • Will follow dietary guidelines, avoiding grapefruit, St. John's wort, and other herbal or unconventional therapies not recommended by the researcher.
      • Will consult with researchers before giving any other medication to the child, considering possible drug interactions.
      • The study does not allow the use of chemotherapy and other targeted drugs at the same time.

Who cannot join the trial?

5 criteria

  • Patients who are not children. This study is only for children.
  • Patients who do not have **Neurofibromatosis type 2**. This is a condition where certain genes do not work properly, leading to the growth of tumors in the nervous system.
  • Patients who are not able to undergo radiological assessments. This means they cannot have imaging tests like X-rays or MRIs to check the size of tumors.
  • Patients who are not able to take the medication **crizotinib**. This is a drug used in the study to see if it affects tumor size.
  • Patients who are not part of the vulnerable population selected for this study. This means the study is focused on a specific group of children who are considered vulnerable.
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Investigated drugs

Crizotinib is a medication being studied for its effectiveness in treating children with severe type 2 neurofibromatosis. This condition involves the growth of tumors in the nervous system, and crizotinib is being tested to see if it can help reduce the size of these tumors, especially in cases where surgery or radiotherapy is not an option.

What is already known about the treatment

Crizotinib – Crizotinib is administered orally in the form of capsules. It is currently being studied in clinical trials for its effectiveness in treating children with severe neurofibromatosis type 2, especially those who cannot undergo surgery or radiotherapy. The main therapeutic indication for crizotinib in this context is to reduce the size of tumors associated with this genetic condition. At the molecular level, crizotinib works by inhibiting specific proteins that are involved in the growth and survival of cancer cells. It is classified as a tyrosine kinase inhibitor, which is a type of targeted cancer therapy.

Investigated diseases

Neurofibromatosis type 2 – Neurofibromatosis type 2 is a genetic disorder caused by a mutation that affects the gene responsible for controlling cell division. This leads to the development of benign tumors in the peripheral nervous system and various benign or locally malignant tumors in the central nervous system. The condition is characterized by the growth of these tumors, which can cause a range of neurological symptoms depending on their location. In children, complications from the disease can occur more frequently than in adults. The progression of the disease involves the potential for multiple tumors to develop over time.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IITrial ID2024-516607-16-00Protocol codeKRONF2Estimated enrolment1 patientSponsorMedical University Of Warsaw

sourced from the EU Clinical Trials Register and site verification

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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).