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Study of Entrectinib in Children and Adolescents with Advanced or Metastatic Solid Tumors or Primary CNS Tumors with No Satisfactory Treatment Options

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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 the effects of a medication called entrectinib, also known by its brand name Rozlytrek, in children and adolescents. The study is specifically for those with advanced or spreading solid tumors or primary tumors in the central nervous system (CNS), which includes the brain and spinal cord. These tumors are challenging to treat, and the study aims to find new treatment options for young patients who have no satisfactory treatments available.

The purpose of the study is to determine the best dose of entrectinib for children and to evaluate how effective it is in treating these tumors. The study will involve taking the medication in different forms, such as capsules or granules, which can be swallowed or given through a feeding tube. The study will also look at how the body processes the medication and any side effects that may occur. The study will assess the response of the tumors to the treatment using specific criteria designed to measure changes in tumor size and growth.

Participants in the study will receive either entrectinib or a placebo, and their health will be closely monitored throughout the trial. The study will help researchers understand how well entrectinib works in treating these types of tumors in children and adolescents, and it will provide valuable information on the safety and effectiveness of the medication. The trial is expected to continue until 2026, with the goal of improving treatment options for young patients with these challenging conditions.

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, the patient will be assessed to ensure they meet the eligibility criteria. This includes being under 18 years of age and having a specific type of tumor with certain genetic characteristics.

    The patient must have a performance status score of at least 60% and a minimum life expectancy of four weeks.

  2. Step 2

    Initial treatment phase

    The patient will begin treatment with entrectinib, which is administered in the form of Rozlytrek capsules or granules. The medication can be taken orally or through a feeding tube.

    The initial phase involves determining the maximum tolerated dose or the recommended dose for the next phase. This is done by monitoring for any dose-limiting side effects during the first 28 days.

  3. Step 3

    Dose confirmation and expansion

    Once the appropriate dose is established, the patient will continue to receive entrectinib at this dose. The study will evaluate the effectiveness of the treatment by measuring the response rate of the tumors.

    The patient's response to the treatment will be assessed using specific criteria for brain and other solid tumors.

  4. Step 4

    Monitoring and assessment

    Throughout the study, the patient will undergo regular monitoring to assess the incidence and severity of any side effects, as well as changes in laboratory and heart function tests.

    The study will also evaluate the concentration of entrectinib in the blood and its effects on the patient's growth, puberty, and neurological function.

  5. Step 5

    Completion of the study

    The study is expected to continue until August 2026. Upon completion, the overall effectiveness and safety of entrectinib will be evaluated based on the collected data.

    The final assessment will include the patient's overall survival and any changes in their condition during the study period.

Who can join the trial?

6 criteria

  • Participants can be male or female and must be under 18 years old.
  • Participants must have a measurable or evaluable disease. This means the disease can be seen or measured using medical tests.
  • The tumors must have specific gene fusions called NTRK1/2/3 or ROS1. Gene fusions are changes in the genes that can affect how cells grow.
  • Participants must have a diagnosis of cancer either at the start or if the cancer has come back.
  • If the participant's tumor was tested locally, a sample of the tumor tissue must be sent for further testing at a special laboratory.
  • Participants must have a performance status score of at least 60%. This score measures how well they can perform daily activities. They must also have a minimum life expectancy of at least 4 weeks.

Who cannot join the trial?

10 criteria

  • Patients who have not fully recovered from the side effects of previous treatments.
  • Patients with other serious health conditions that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent major surgery.
  • Patients with certain types of infections that are not well controlled.
  • Patients who are pregnant or breastfeeding.
  • Patients who have allergies to the study medication or similar drugs.
  • Patients who have certain heart conditions that are not stable.
  • Patients who have a history of certain mental health conditions that are not well managed.
  • Patients who are unable to follow the study procedures or instructions.
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Investigated drugs

Entrectinib (RXDX-101) is a medication being studied for its potential to treat pediatric patients with locally advanced or metastatic solid tumors, or primary central nervous system (CNS) tumors. It is particularly focused on patients who have no satisfactory treatment options available. The trial aims to determine the most effective and safe dose of entrectinib for children, whether they can swallow capsules or need the medication administered through a feeding tube. The study also evaluates how well the medication works in shrinking tumors that have specific genetic changes, known as NTRK1/2/3 or ROS1 gene fusions. The effectiveness of the treatment is assessed using specific criteria for measuring tumor response.

What is already known about the treatment

Entrectinib – Entrectinib is administered orally, either as capsules or minitablets, and can also be given through a feeding tube for those unable to swallow. It is currently being studied in clinical trials for its effectiveness in treating pediatric patients with advanced or metastatic solid tumors and primary central nervous system tumors, especially those with specific genetic mutations. The medication works by targeting and inhibiting certain proteins, specifically those produced by NTRK1/2/3 or ROS1 gene fusions, which are involved in the growth and survival of cancer cells. Entrectinib is classified as a targeted cancer therapy, focusing on specific molecular targets associated with cancer.

Investigated diseases

  • Pediatric Solid Tumors

    These are abnormal masses of tissue that can occur in various parts of a child's body, such as bones, muscles, or organs. They develop when cells grow uncontrollably and form a lump or mass. The progression of these tumors can vary widely depending on their location and type. Some may grow slowly and remain localized, while others can grow rapidly and spread to other parts of the body. Symptoms often depend on the tumor's size and location, potentially causing pain, swelling, or functional impairment. Early detection and monitoring are crucial for managing these tumors effectively.

  • Primary Central Nervous System (CNS) Tumors

    These tumors originate in the brain or spinal cord and can affect children. They arise from various types of cells within the central nervous system and can be either benign or malignant. The progression of CNS tumors can lead to increased pressure within the skull, causing headaches, nausea, or changes in behavior. Depending on their location, they may also affect movement, balance, or cognitive functions. The growth rate and impact of these tumors can vary, necessitating careful observation and management.

Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase ITrial ID2023-505088-35-00Protocol codeCO40778Estimated enrolment93 patientsSponsorF. Hoffmann-La Roche AG

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).