Ongoing Clinical Trials for Inflammatory Myofibroblastic Tumour
There are currently 2 clinical trials investigating new treatment options for inflammatory myofibroblastic tumours. These studies are evaluating targeted medications called tyrosine kinase inhibitors that work by blocking specific proteins involved in tumour growth. Both trials are being conducted across multiple European countries and focus on patients with genetic changes in certain genes, particularly ALK (anaplastic lymphoma kinase). The trials include children, teenagers, and young adults who have tumours that cannot be surgically removed or have not responded to standard treatments.
Clinical trial locations
- Austria
- Belgium
- Czechia
- Denmark
- Finland
- France
- Germany
- Italy
- Netherlands
- Poland
- Spain
- Sweden
Study of Brigatinib for Children and Young Adults with ALK+ Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumors, or Other Solid Tumors
This trial is testing a medication called brigatinib in children and young adults who have inflammatory myofibroblastic tumours or other specific types of tumours that test positive for a change in the ALK gene. Brigatinib is a tyrosine kinase inhibitor, which means it works by blocking specific proteins that help tumour cells grow and spread.
Who can join this trial:
- Patients must be at least 1 year old and younger than 26 years at the time of joining
- Patients need to weigh at least 10 kg and be able to swallow tablets
- Patients must have a confirmed diagnosis with test results showing a change in the ALK gene in their tumour, which can be confirmed by different tests such as IHC, FISH, PCR, or NGS
- For patients with inflammatory myofibroblastic tumours, they should not be able to have surgery without causing severe harm, or they may have newly diagnosed advanced tumours where surgery is not possible due to the tumour’s location
- Patients must have a certain level of physical ability, with a Karnofsky performance status of at least 50% for patients older than 16 years, or a Lansky Play Scale of at least 50% for patients 16 years or younger
- Patients must not be taking any other experimental medications or treatments, including herbal therapies, within 30 days before starting the study medication
Who cannot join:
- Patients without a diagnosis of the specified tumour types
- Patients outside the specified age range
- Patients who are part of vulnerable populations that may need special protection
What the trial involves:
The trial is studying brigatinib, which comes as film-coated tablets in doses of 30 mg, 90 mg, and 180 mg, taken by mouth. The study has several phases. First, researchers will determine the most suitable dosage for young patients by identifying any dose-limiting side effects and understanding how the body processes the medication. This dosage will be adjusted for younger patients based on how similar it is to adult doses. A board of experts will make the final decision on the recommended dose.
The effectiveness of brigatinib will be evaluated by measuring tumour size and monitoring any changes in the patient’s condition. The study will assess the overall response rate, which is the percentage of patients who experience a significant reduction in tumour size or complete disappearance of the tumour. Treatment is planned to continue for up to two years, provided patients continue to benefit without significant side effects. The trial is expected to continue until 2030.
Study of Crizotinib and Temsirolimus for Children with ALK, ROS1, or MET Positive Cancers, Including Neuroblastoma and Rhabdomyosarcoma
This trial is testing crizotinib, either alone or in combination with another medication called temsirolimus, for children with inflammatory myofibroblastic tumours and other types of tumours that have specific genetic markers such as ALK, ROS1, or MET. Crizotinib is a tyrosine kinase inhibitor that targets specific proteins in tumour cells to help stop their growth and spread. Temsirolimus works by blocking a protein that helps tumour cells grow.
Who can join this trial:
- Patients must be between 1 and 21 years old
- Patients must have a confirmed diagnosis of inflammatory myofibroblastic tumours or other specified types of tumours that have returned after treatment or not responded to standard treatments
- Patients must have specific genetic changes in their tumour, such as changes in the ALK, MET, or ROS1 genes, which can be detected through special tests
- Patients must have a performance status score greater than 60%, which measures how well they can perform daily activities
- Patients must have a life expectancy of at least 12 weeks
- Patients must have measurable disease, meaning the tumour can be measured in size using medical imaging
- Patients must have completed any previous treatments at least 2 weeks before starting the study medication and must not have had any previous treatments that directly target the ALK, ROS1, or MET genes
- Patients 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:
- Patients with a different type of tumour that is not listed in the study
- Patients outside the specified age range
- Patients who are pregnant or breastfeeding
- Patients currently participating in another clinical trial
- Patients with medical conditions that could interfere with the study treatment, including certain heart conditions or uncontrolled infections
- Patients with allergies to the study medications
- Patients with a history of drug or alcohol abuse
- Patients who are unable to follow study procedures or provide consent
What the trial involves:
Upon joining the study, patients undergo an initial assessment to confirm eligibility, including a review of their medical history and current health status. Treatment begins with crizotinib, which is provided as capsules in doses of 200 mg and 250 mg, taken by mouth as prescribed. For certain patients, crizotinib is combined with temsirolimus, which is given through an intravenous infusion. The combination aims to determine the recommended dose and assess safety and effectiveness in tumours with the specific genetic markers.
Throughout the trial, regular monitoring is conducted to assess response to treatment, including measuring tumour size and evaluating overall health. The study will observe dose-limiting side effects and overall response rates, as well as progression-free survival and overall survival. After completing treatment cycles, follow-up assessments are scheduled to monitor long-term effects and health outcomes. The trial is expected to continue until December 31, 2029.
Summary
Both ongoing clinical trials for inflammatory myofibroblastic tumours focus on targeted therapies that block specific proteins driving tumour growth. These studies are part of a broader effort to develop more effective treatments for rare tumours in children and young adults.
The trials are being conducted across multiple European countries, with the brigatinib study running in 12 countries including Sweden, Poland, Belgium, France, Finland, Germany, Italy, Spain, Denmark, Czechia, Austria, and the Netherlands. The crizotinib and temsirolimus study is being conducted in 6 countries: Spain, Italy, Germany, France, Denmark, and the Netherlands.
A notable feature of both trials is their focus on patients whose tumours have specific genetic changes, particularly in the ALK gene. These genetic markers help identify which patients are most likely to benefit from these targeted treatments. Both studies require that patients have tumours that cannot be surgically removed without causing severe harm or have not responded to standard treatments.
The trials differ in their approach: the brigatinib study focuses on a single medication taken as tablets, while the crizotinib study tests the medication both alone and in combination with temsirolimus. The combination approach may provide additional treatment options for patients whose tumours do not respond adequately to single-agent therapy. Both trials have long timelines, extending to 2029 or 2030, which will allow researchers to gather comprehensive data on the long-term safety and effectiveness of these treatments.
