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Study of Crizotinib and Temsirolimus for Children with ALK, ROS1, or MET Positive Cancers, Including Neuroblastoma and Rhabdomyosarcoma

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 clinical trial is focused on studying certain types of cancer in children, specifically those that are positive for ALK, ROS1, or MET genes. The cancers being studied include Neuroblastoma, Rhabdomyosarcoma, Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumors, and other related malignancies. The treatment being tested is a medication called Crizotinib, which may be used alone or in combination with another drug called Temsirolimus. Crizotinib is also known by its code name PF-02341066 and is available in different forms, such as capsules and oral solutions.

The purpose of this study is to find the best dose of Crizotinib when used with Temsirolimus and to assess the safety and initial effectiveness of Crizotinib on its own in treating these specific gene-positive tumors. Participants in the study will receive either Crizotinib alone or in combination with Temsirolimus, depending on their specific cancer type and genetic markers. The study will monitor how the body responds to the treatment and any side effects that may occur.

Throughout the study, participants will undergo regular check-ups and tests to track their progress and the impact of the treatment on their cancer. The study aims to gather important information that could help improve treatment options for children with these types of cancers in the future. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the actual treatment. The study is expected to continue until the end of 2029.

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

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and current health status.

    Eligibility criteria include a confirmed diagnosis of specific cancers such as neuroblastoma, rhabdomyosarcoma, or other ALK, ROS1, or MET positive malignancies. Age must be between 1 and 21 years, with a performance status score above 60%.

  2. Step 2

    Treatment initiation

    The treatment phase begins with the administration of crizotinib as a single agent or in combination with temsirolimus.

    Crizotinib is provided in the form of hard capsules or granules for oral consumption. Dosages include 200 mg and 250 mg capsules, taken as prescribed by the study team.

  3. Step 3

    Combination therapy

    For certain patients, crizotinib is combined with temsirolimus, which is administered through an intravenous infusion.

    The combination aims to determine the recommended dose and assess safety and preliminary activity in tumors positive for ALK, MET, or ROS1.

  4. Step 4

    Monitoring and evaluation

    Throughout the trial, regular monitoring is conducted to assess the response to treatment. This includes measuring tumor size and evaluating overall health.

    Primary endpoints include observing dose-limiting toxicities and overall response rates. Secondary endpoints involve progression-free survival and overall survival.

  5. Step 5

    Follow-up

    After completing the treatment cycles, follow-up assessments are scheduled to monitor long-term effects and overall health outcomes.

    The trial is expected to continue until December 31, 2029, with ongoing evaluations to ensure patient safety and treatment efficacy.

Who can join the trial?

10 criteria

  • The patient must have a confirmed diagnosis of certain types of cancer, such as Neuroblastoma (NBL), Rhabdomyosarcoma (RMS), Anaplastic Large Cell Lymphoma (ALCL), or Inflammatory Myofibroblastic Tumors (IMT). These cancers must have returned after treatment or not responded to standard treatments.
  • The patient must be between 1 and 21 years old.
  • The patient must have a performance status score greater than 60%. This score measures how well the patient can perform daily activities.
  • The patient must have specific genetic changes in their cancer, such as changes in the ALK, MET, or ROS1 genes. These changes can be detected through special tests.
  • The patient must have a life expectancy of at least 12 weeks.
  • The patient must have measurable disease, meaning the cancer can be measured in size using medical imaging.
  • The patient must have completed any previous cancer treatments at least 2 weeks before starting the study medication.
  • The patient must not have had any previous treatments that directly target the ALK, ROS1, or MET genes.
  • The patient must not have taken any other investigational drugs or had major surgery in the past 2 weeks.
  • Male and female patients who can have children must agree to use effective birth control methods during the study.

Who cannot join the trial?

12 criteria

  • Patients who have a different type of cancer that is not listed in the study.
  • Patients who are not within the specified age range for the study.
  • Patients who are not able to follow the study procedures or instructions.
  • Patients who have a medical condition that could interfere with the study treatment.
  • Patients who are pregnant or breastfeeding.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent surgery or medical procedure that could affect the study.
  • Patients who have an allergy or reaction to the study medications.
  • Patients who have a history of certain heart conditions.
  • Patients who have an infection that is not well controlled.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who are unable to provide consent to participate in the study.
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Investigated drugs

  • Crizotinib

    is a medication used in this trial to treat certain types of cancer. It works by targeting specific proteins in cancer cells, which can help stop the growth and spread of the cancer. In this study, crizotinib is being tested both on its own and in combination with another medication to see how effective it is in treating cancers that have specific genetic markers, such as ALK, ROS1, or MET.

  • Temsirolimus

    is another medication used in this trial. It is being combined with crizotinib to see if the combination is more effective than crizotinib alone. Temsirolimus works by blocking a protein that helps cancer cells grow, which may help slow down or stop the progression of the cancer. The study aims to find the best dose of this combination and to evaluate its safety and effectiveness in treating certain types of cancer in children.

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 certain types of cancer, specifically those with ALK, ROS1, or MET gene alterations. The main therapeutic indications include neuroblastoma, rhabdomyosarcoma, anaplastic large cell lymphoma, and inflammatory myofibroblastic tumors. Crizotinib works by inhibiting the activity of specific proteins that promote cancer cell growth, thereby slowing or stopping the progression of the disease. It is classified as a tyrosine kinase inhibitor, a type of targeted cancer therapy.

Investigated diseases

  • Neuroblastoma

    A cancer that develops from immature nerve cells found in several areas of the body, most commonly in and around the adrenal glands. It primarily affects children and can also occur in the abdomen, chest, neck, and near the spine. The disease often begins in the early stages of development and can spread to other parts of the body, including the bones and bone marrow.

  • Rhabdomyosarcoma

    A type of cancer that forms in soft tissue, such as muscle, and can occur in any part of the body. It is most common in children and can develop in areas like the head, neck, bladder, and reproductive organs. The disease progresses by forming tumors that can invade nearby tissues and spread to other parts of the body.

  • Anaplastic Large Cell Lymphoma

    A type of non-Hodgkin lymphoma that involves the T-cells, which are part of the immune system. It is characterized by large, abnormal lymphocytes and can affect lymph nodes and other organs. The disease can progress rapidly, leading to symptoms such as swollen lymph nodes, fever, and weight loss.

  • Inflammatory Myofibroblastic Tumors

    Rare tumors that can occur in various parts of the body, including the lungs, abdomen, and pelvis. These tumors are composed of myofibroblastic spindle cells and inflammatory cells. They can cause symptoms depending on their location and may grow slowly or more aggressively.

  • Other ALK/ROS1/MET-positive malignancies

    These are cancers that have genetic alterations in the ALK, ROS1, or MET genes, which can drive cancer growth. Such malignancies can occur in various tissues and organs, leading to diverse symptoms based on the affected area. The progression of these cancers can vary, with some being more aggressive than others.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-504880-18-00Protocol codeCRISP ITCC-053Estimated enrolment82 patientsSponsorErasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

sourced from the EU Clinical Trials Register and site verification

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