Medulloblastoma

This study tests a drug called ulixertinib in children and teens whose advanced cancers have a specific genetic change in the MAPK pathway. The goal is to see if the drug can shrink or control the tumor. About 20 participants with various solid tumors, lymphomas, or related disor…

This study tests a combination of a vaccine targeting the CMV virus (PEP-CMV) and an immunotherapy drug (nivolumab) in children and young adults with certain aggressive brain tumors, including diffuse midline glioma and medulloblastoma. The goal is to see if this approach is safe…

This Phase I trial tests whether infusing specially trained immune cells, called TAA-T, can safely target and attack high-risk brain tumors in children and adults. The cells are made from the patient's own blood and are designed to recognize multiple tumor-related proteins, poten…

This phase 2 trial tests the drug selpercatinib in children and young adults (ages 1 to 21) with advanced cancers that have a specific genetic change called a RET alteration. The goal is to see if the drug can shrink tumors or stop them from growing. Only one participant has been…

This early-phase study tests a flavored, drinkable version of the chemotherapy drug irinotecan (VAL-413) combined with another drug (temozolomide) in children and young adults up to age 30 whose solid tumors have come back. The main goal is to find the safest dose and see how the…

This trial tests the drug savolitinib in children and adults whose brain tumors (medulloblastoma, high-grade glioma, or DIPG) have come back or not responded to treatment. The drug works by blocking an enzyme that some tumor cells need to grow. The study aims to find the safest d…

This Phase 2 trial tests a new approach for children whose medulloblastoma or ependymoma has come back. It combines a radioactive antibody (omburtamab) delivered directly into the brain's fluid spaces with standard chemotherapy drugs. The goal is to see if this combination can he…

This early-stage trial is testing the safety and potential benefit of pembrolizumab, an immunotherapy drug, in 71 children whose brain tumors (like DIPG, high-grade gliomas, or medulloblastoma) have come back or not responded to standard treatments. The drug works by helping the …

This phase 2 trial is testing whether giving lower doses of radiation and chemotherapy can effectively treat a specific type of brain tumor called WNT-driven medulloblastoma in children and young adults aged 3 to 21. The goal is to kill cancer cells while reducing long-term side …

This study tests a drug called tipifarnib in children and young adults (ages 1 to 21) whose cancers have a specific change in the HRAS gene. The cancers are advanced or have come back after treatment. The goal is to see if tipifarnib can shrink tumors or stop them from growing. A…

This study tested the drug ensartinib in 13 children and young adults (ages 1–21) whose cancers had come back or did not respond to treatment and had specific ALK or ROS1 gene changes. The goal was to see if the drug could shrink or stop tumor growth. While the drug targets cance…

This early-stage trial tests a new treatment for children and young adults up to age 21 with certain brain tumors that have come back or not responded to standard therapy. The treatment uses the patient's own immune cells, which are modified in a lab to recognize and attack a pro…

This phase 2 trial tested the drug ivosidenib in children and young adults (ages 1 to 21) with solid tumors, lymphomas, or histiocytic disorders that have an IDH1 mutation and have either come back after treatment or not responded. The goal was to see if the drug could shrink or …

This study tests a drug called entrectinib in children with advanced solid tumors or brain tumors that have come back or haven't responded to other treatments. The trial has two parts: first finding the best dose, then seeing how well it works in children whose tumors have specif…

This study tests a drug called erdafitinib in children and young adults (ages 1 to 21) whose cancers have come back or are not responding to treatment and have specific changes in FGFR genes. The drug works by blocking enzymes that help cancer cells grow. The main goal is to see …

This early-stage trial tests a vaccine called PEP-CMV in 30 children and young adults (ages 3–35) whose medulloblastoma or malignant glioma has come back. The vaccine is designed to train the immune system to attack tumor cells that carry a common virus protein (CMV). Participant…

This phase 2 trial is testing the drug larotrectinib in children and young adults (ages 1 to 21) with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have a specific genetic change called an NTRK fusion. The cancers have either come back after treatment…

This early-phase study tests a vaccine called SurVaxM in 35 children with certain brain tumors that have come back or are hard to treat. The vaccine is designed to teach the immune system to find and destroy tumor cells that have a protein called survivin. The goal is to see if i…

This study tests whether giving three rounds of very strong chemotherapy with stem cell rescue works better than one round for children under 10 with high-risk brain tumors. The goal is to improve survival without adding too many side effects. About 250 children will take part.

This clinical trial tests a risk-adapted approach for young children with newly diagnosed brain tumors. The treatment starts with chemotherapy to delay radiation, giving the brain more time to develop. Then, radiation is carefully targeted to the tumor area to reduce side effects…

This early-stage trial tests a radioactive drug called CLR 131 in 30 children and young adults with solid tumors, lymphoma, or brain tumors that have come back or not responded to standard treatments. The main goals are to find a safe dose and see if the drug can shrink tumors. P…

This early-phase trial tests a new treatment for adults with aggressive brain cancers (glioblastoma, ependymoma, or medulloblastoma) that have spread to the lining of the brain and spinal cord. The treatment uses the patient's own immune cells, modified in a lab to recognize and …

This study tests a daily combination of sirolimus, celecoxib, and low-dose chemotherapy in 46 children whose solid or brain tumors have returned or not responded to standard treatment. The goal is to see if this approach can shrink tumors or stop them from growing. While not a cu…

This study aimed to test a new way to treat brain tumors that came back after standard therapy. The plan was to deliver immunotherapy (nivolumab) plus chemotherapy directly into the fluid spaces of the brain in children and adults. However, the study was withdrawn before any part…

This study tests whether adding an experimental drug called indoximod to standard chemotherapy and radiation can help children aged 3 to 21 with certain brain cancers. The drug aims to boost the body's immune system to fight tumors more effectively. The trial includes children wi…

This early-phase study tests whether specially engineered immune cells (CAR T cells) can be safely given directly into the brain or spinal fluid of children and young adults with HER2-positive brain tumors that have come back or not responded to standard treatments. The therapy a…

This clinical trial is testing whether lower doses of radiation and chemotherapy can effectively treat children with medulloblastoma, a type of brain tumor, while reducing long-term side effects. The study includes 360 children aged 3 to 21 years with specific tumor types. Resear…

This phase 2 trial tests a personalized approach to treating medulloblastoma, a type of brain cancer. Instead of using a one-size-fits-all treatment, the study assigns radiation and chemotherapy doses based on the tumor's molecular subtype and the patient's risk level. The goal i…

This study tests whether a special PET scan using a radioactive form of methionine (a natural amino acid) can help doctors see tumors in children and young adults with various cancers, including brain tumors and sarcomas. About 503 participants will be scanned to see if the metho…

This study tests whether a new type of PET/CT scan, using a radioactive tracer called 68Ga-DOTATOC, can find neuroendocrine tumors (NETs) and similar tumors more safely and accurately than current imaging methods. About 800 people with known or suspected NETs will receive the sca…

This study compares three different PET/CT scans (68Ga-HA-DOTATATE, 18F-DOPA, and 18F-FDG) in 50 people with metastatic neuroendocrine tumors. The goal is to see if all tumors show up on the standard scan or if some are missed, which could change treatment. Participants will get …

This study tests a special proton radiation method for children with brain tumors that need radiation to the brain and spine. The new approach spares parts of the backbone to reduce side effects like low blood counts and slowed bone growth. About 20 children aged 3 to 18 will rec…

This study investigates whether the emotional distress of parents—such as depression, anxiety, or trauma—affects how well children with recurrent medulloblastoma follow their treatment plans. Researchers will follow 450 parent-child pairs across four hospitals, measuring parental…

This study screens children and teens with advanced solid tumors, lymphomas, or histiocytic disorders to find genetic changes in their cancer cells. Participants undergo biopsies and scans to identify mutations that can be targeted by specific drugs. The goal is to see if matchin…

This study looks at how surviving a pediatric brain tumor affects thinking and behavior. Researchers will use brain scans to examine brain structure in children who have completed treatment. The goal is to better understand the long-term cognitive challenges these survivors may f…

This study uses a special PET scan to measure brain inflammation in children with medulloblastoma who receive whole-brain radiation. Twenty-two participants will be scanned four times over two years to see how inflammation changes. The goal is to understand how radiation affects …

This pilot study is testing a radioactive antibody called 124I-Humanized 3F8 to see how it travels through the body and attaches to tumors in people with melanoma, neuroblastoma, or sarcoma. Seven participants will receive the antibody and then have PET/CT scans at several time p…