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

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What is this study about?

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.

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 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 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 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 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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Oncopole Claudius Regaud	Toulouse	France

Other Sites

Site Name	City	Country
St. Olavs Hospital HF	Trondheim	Norway
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Narodny Ustav Detskych Chorob	Bratislava	Slovakia
Virgen del Rocío University Hospital	Sevilla	Spain
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Assistance Publique Hopitaux De Paris	Paris	France
Rigshospitalet	Copenhagen	Denmark
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Unlijbpxow Mdbmzhi Clcjvj Hjvicoxocdkixhcbp	Hamburg	Germany
Uvgdjbvjxcsgadkhtgldv Epbfz Ahh	Essen	Germany
Hkopbytj Unnsgdrwft Cyqfoem Hstocpav	Helsinki	Finland
Acilrvt Ogdxnptvbcm Uiqluichtoxqc Ciszpuxtgfnw Ducug Seakrb E Dvlgs Smfhtcc Dy Tmdkkx	Turin	Italy
Uincusnjvauvaqckpvthf Mohodubg Aqu	Munster	Germany
Czic Dr Npwep	Vandoeuvre Les Nancy	France
Gvksrl Uuramwcgkw Fiinziyxp	Frankfurt	Germany
Hlxcdhdh Vkxu dzotwrut	Barcelona	Spain
Chxnuf Onzxk Liwdzcw	Lille	France
Iwiiwfbe Cjdgo	Paris	France

Trial status

Country	Status	Recruitment Start
Denmark	Recruiting	18.04.2018
Finland	Recruiting	18.04.2018
France	Recruiting	18.04.2018
Germany	Recruiting	18.04.2018
Italy	Not recruiting	18.04.2018
Norway	Recruiting	18.04.2018
Slovakia	Not yet recruiting	18.04.2018
Spain	Recruiting	18.04.2018
The Netherlands	Recruiting	18.04.2018

Trial locations

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.

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 ID:

2023-504880-18-00

Protocol code:

CRISP ITCC-053

Trial Phase:

Human Pharmacology (Phase I) – Other

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

What is this study about?

Who Can Join the Study?

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