Clinical Trials for Central Nervous System Neoplasms

Currently, there are 5 ongoing clinical trials testing new treatments for patients with central nervous system neoplasms. These studies are taking place across multiple European countries and are investigating targeted therapies and immunotherapies for various types of brain and spinal cord tumors, particularly in children and young adults. The trials focus on treatments that target specific genetic changes in tumor cells, including MET, NTRK, and RET alterations, as well as somatostatin receptor-positive tumors.

Clinical trial locations

• Czechia
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
• Denmark
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
  • Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors
• France
  • Study on APL-101 for Treating Advanced Solid Tumors and Non-Small Cell Lung Cancer with MET Changes in Patients
  • Study of Avelumab and Lenvatinib for Children with Central Nervous System Tumors
  • Study to Find the Right Dose and Safety of Lutetium (177Lu) Edotreotide and Arginine-Lysine in Children with Somatostatin Receptor-Positive Tumors
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
  • Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors
• Germany
  • Study of Avelumab and Lenvatinib for Children with Central Nervous System Tumors
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
  • Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors
• Hungary
  • Study on APL-101 for Treating Advanced Solid Tumors and Non-Small Cell Lung Cancer with MET Changes in Patients
• Ireland
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
• Italy
  • Study on APL-101 for Treating Advanced Solid Tumors and Non-Small Cell Lung Cancer with MET Changes in Patients
  • Study to Find the Right Dose and Safety of Lutetium (177Lu) Edotreotide and Arginine-Lysine in Children with Somatostatin Receptor-Positive Tumors
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
  • Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors
• Netherlands
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
• Spain
  • Study on APL-101 for Treating Advanced Solid Tumors and Non-Small Cell Lung Cancer with MET Changes in Patients
  • Study to Find the Right Dose and Safety of Lutetium (177Lu) Edotreotide and Arginine-Lysine in Children with Somatostatin Receptor-Positive Tumors
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion
  • Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors
• Sweden
  • Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion

Study on APL-101 for Treating Advanced Solid Tumors and Non-Small Cell Lung Cancer with MET Changes in Patients

This trial is testing APL-101, also known as Bozitinib, for treating advanced tumors that have specific changes in a gene called MET. While the study focuses primarily on non-small cell lung cancer, it also includes patients with tumors in the brain and spinal cord that show MET alterations.

Main inclusion criteria: Participants must be at least 18 years old and able to provide informed consent. They should have advanced or spreading solid tumors that cannot be removed surgically and have not responded to standard treatments. Patients must have at least one measurable tumor and acceptable organ and heart function. Women who can become pregnant must have a negative pregnancy test and agree to use effective birth control. Patients must have an expected survival of at least 3 months.

Main exclusion criteria: The trial excludes patients with cancer types not being studied, those without MET gene changes, and individuals unable to follow study procedures. Patients with serious health conditions that could interfere with the study, pregnant or breastfeeding women, and those currently in other clinical trials are not eligible. People who have had severe allergic reactions to similar medications or have not recovered from previous cancer treatment side effects are also excluded.

Trial focus: The study evaluates the safety and effectiveness of APL-101 in treating tumors with MET genetic changes. Participants receive APL-101 as an oral capsule with regular monitoring through check-ups, blood tests, and imaging studies to track tumor size and side effects. The trial aims to determine the best dose and assess how well the medication works against these specific tumor types.

Investigational drug: APL-101 is taken by mouth and works by targeting and inhibiting the MET protein, which plays a role in cell growth and survival. This targeted therapy is designed to slow down or stop cancer progression in tumors with MET alterations.

Study of Avelumab and Lenvatinib for Children with Central Nervous System Tumors

This clinical trial tests a combination of two medications, Avelumab and Lenvatinib, in children with brain or spinal cord tumors. The study aims to determine whether these medications are safe and effective when used together.

Main inclusion criteria: Children between 2 and 18 years old can participate. They must have a confirmed diagnosis of a primary brain or spinal cord tumor that is high-grade and has been treated with radiotherapy and at least one previous treatment. Participants need measurable disease on scans and a performance status score of at least 50. Girls who have started menstruating must have a negative pregnancy test. Children must have adequate bone marrow, kidney, liver, and heart function. Those with controlled seizures can participate if their medication is stable.

Main exclusion criteria: The trial excludes children with other types of cancer, those who have not recovered from previous treatments, and patients with severe heart problems or uncontrolled high blood pressure. Children with active infections, pregnant or breastfeeding girls, and those with severe allergic reactions to similar medications cannot participate. Individuals with certain autoimmune diseases or those currently in another clinical trial are also excluded.

Trial focus: The study is divided into two parts. The first part determines the safest dose for children, while the second part evaluates how well the combination works in treating tumors. Avelumab is given through an intravenous infusion, while Lenvatinib is taken as oral capsules. Regular monitoring includes imaging tests and blood tests to assess response and side effects.

Investigational drugs: Avelumab is an immune checkpoint inhibitor that helps the immune system recognize and attack cancer cells. Lenvatinib targets certain proteins in cancer cells to stop tumor growth and spread. Together, they are being tested to see if the combination is more effective than either drug alone.

Study to Find the Right Dose and Safety of Lutetium (177Lu) Edotreotide and Arginine-Lysine in Children with Somatostatin Receptor-Positive Tumors

This trial studies a targeted radiopharmaceutical therapy called lutetium Lu 177 edotreotide for children with tumors that have somatostatin receptors. These special receptors on tumor cells allow the treatment to deliver radiation directly to cancer cells.

Main inclusion criteria: Children must be between 2 and 18 years old with a confirmed diagnosis of somatostatin receptor-positive tumors, which can include brain and spinal cord tumors. The tumor must have returned or not responded to at least one previous treatment. Participants need a performance score of at least 50 and must have recovered from previous treatment side effects. Heart function must be adequate if previous treatments included certain chemotherapy or chest radiation. Scans must show that tumors take up more radioactivity than the liver, and all important tumor areas must be positive for somatostatin receptors.

Main exclusion criteria: Children under 2 years old cannot participate. Those without somatostatin receptor-positive tumors or who have not tried at least one other treatment are excluded. Pregnant or breastfeeding girls, patients unable to safely receive the treatment, and those unable to follow study procedures are not eligible.

Trial focus: The study aims to determine the appropriate dose of lutetium Lu 177 edotreotide for children based on safety and how the body processes the therapy. The treatment is given through an intravenous infusion, delivering radiation directly to tumor cells while minimizing damage to healthy tissue. An arginine-lysine solution is also given to protect the kidneys during treatment. Regular monitoring includes imaging tests and blood tests to assess response and side effects.

Investigational drug: Lutetium Lu 177 edotreotide is a radiopharmaceutical that binds to somatostatin receptors on tumor cells, delivering targeted radiation to destroy cancer cells while sparing healthy tissue.

Study on Larotrectinib for Treating Children with Advanced Solid Tumors with NTRK Fusion

This trial investigates larotrectinib, an oral medication for children with tumors that have a specific genetic change called NTRK fusion. The study includes patients with brain and spinal cord tumors that have this genetic alteration.

Main inclusion criteria: Patients from birth to 21 years old with solid tumors or brain and spinal cord tumors that have an NTRK fusion can participate. The tumors must have returned, worsened, or not responded to other treatments with no other standard options available. Patients must have adequate blood cell counts, liver, and kidney function. They need a performance score of at least 50 and must be able to swallow capsules or take medicine through a tube. Those who can have children must agree to use effective birth control.

Main exclusion criteria: Patients without solid tumors with NTRK fusion cannot participate. Those outside the specified age range, unable to take oral medication, or with other medical conditions that might interfere with the study are excluded. Pregnant or breastfeeding individuals, those in another clinical trial, and patients who have not recovered from previous treatments are not eligible.

Trial focus: The study is divided into two phases. Phase 1 determines the safety of larotrectinib and identifies side effects, while Phase 2 evaluates how well tumors respond to treatment. Participants receive larotrectinib orally as a capsule or solution with regular monitoring to measure drug concentration in the blood and assess tumor response, overall health, and quality of life.

Investigational drug: Larotrectinib works by inhibiting TRK proteins that are involved in the growth of cancer cells with NTRK fusions. This targeted therapy aims to shrink or control tumor growth in children with these specific genetic changes.

Study on the Effects of Selpercatinib in Children with Advanced RET-Altered Solid Tumors or Primary Central Nervous System Tumors

This clinical trial studies Selpercatinib in children and young adults with advanced tumors that have changes in the RET gene. The trial includes patients with brain and spinal cord tumors showing these specific genetic alterations.

Main inclusion criteria: Patients between 12 and 21 years old with solid tumors or brain and spinal cord tumors that have returned, worsened, or not responded to available treatments can participate. This includes patients who would need major surgery to remove the tumor. Participants must have adequate blood health, liver, pancreas, and kidney function. They need to be able to follow the treatment plan and attend clinic visits. Those who can have children must use two effective birth control methods. Patients must be able to swallow capsules or take medicine through a tube. There must be evidence of RET gene changes in the tumor or blood. Patients with brain tumors must have stable neurological symptoms and not need increasing steroid doses. A performance score of at least 50 is required.

Main exclusion criteria: Patients without advanced RET-altered tumors, those outside the specified age range, and individuals unable to take Selpercatinib due to allergies or other medical reasons are excluded. Those with other medical conditions that might interfere with the study, currently in another clinical trial, pregnant or breastfeeding, or with a history of certain heart conditions cannot participate. Patients who have not recovered from previous treatments or surgeries are also excluded.

Trial focus: The study is divided into two phases. Phase 1 determines the safety of Selpercatinib and identifies side effects. Phase 2 evaluates how well tumors respond to treatment by measuring tumor size. Participants take Selpercatinib orally as a tablet or solution with regular monitoring through physical exams, blood tests, and imaging studies. The trial aims to find the safest and most effective dose for treating these specific tumor types.

Investigational drug: Selpercatinib is a RET inhibitor that targets and blocks the RET protein, which can contribute to tumor growth when altered. This oral medication aims to slow down or stop the growth of cancer cells in tumors with RET gene changes.

Summary

These five clinical trials represent important research efforts in developing new treatments for brain and spinal cord tumors. Most trials focus on pediatric patients, reflecting the particular need for effective treatments in children with these challenging conditions. The studies are widely distributed across Europe, with France participating in all five trials, followed by Italy and Spain with four trials each, and Germany with three trials.

A notable feature of these studies is their focus on precision medicine, targeting specific genetic changes in tumors. Four of the five trials test medications that work against particular genetic alterations: MET changes, NTRK fusions, RET alterations, and somatostatin receptors. This approach represents a shift toward personalized treatment based on the molecular characteristics of individual tumors rather than just their location or appearance.

The trials involve different types of treatments, including oral medications, intravenous infusions, and radiopharmaceutical therapy. Most studies are designed in two phases, first establishing safety and appropriate dosing, then evaluating effectiveness in controlling or shrinking tumors. Regular monitoring through imaging studies, blood tests, and clinical assessments is a common feature across all trials to ensure patient safety and track treatment response.