Medulloblastoma

This early-phase trial tests a personalized RNA vaccine for people with recurrent medulloblastoma, a type of brain tumor. The vaccine is made from the patient's own tumor material and aims to train the immune system to fight the cancer. The study includes up to 24 participants ag…

This study tests a personalized treatment plan for children under 5 with a type of brain cancer called medulloblastoma. Doctors use the tumor's genetic makeup and the child's age to choose the best mix of chemotherapy and, if needed, radiation. The goal is to improve survival whi…

This early-phase trial tests a new treatment for children and young adults with medulloblastoma or similar brain tumors that have come back or not responded to standard therapy. The treatment uses the patient's own immune cells, modified to recognize and attack tumor cells, deliv…

This study tests a combination of drugs given in low, frequent doses to slow or stop the growth of recurrent brain tumors in children. The goal is to extend survival while maintaining quality of life. About 232 children with medulloblastoma, ependymoma, ATRT, or other rare CNS tu…

This early-stage trial tests whether using focused ultrasound with microbubbles can temporarily open the blood-brain barrier, allowing the immunotherapy drug nivolumab to reach brain tumors more effectively. It includes 30 children and young adults with relapsed diffuse midline g…

This early-stage trial tests whether a specially engineered herpes virus (G207) is safe to inject directly into the brain tumors of children whose cancer has returned. Up to 24 children aged 3 to 21 will receive the virus, and some will also get a small dose of radiation to help …

This early-phase study tests the safety of a new cell therapy called B7-H3 CAR T cells for children and young adults whose brain tumors have come back or are growing despite standard treatment. The therapy uses the patient's own immune cells, modified to target a protein (B7-H3) …

This study tests whether digoxin, a heart medication, can help children and young adults with a specific type of relapsed brain cancer called non-WNT/non-SHH medulloblastoma. About 23 participants aged 1 to 29 will receive digoxin to see if it stops the cancer from growing for at…

This study tests whether sodium thiosulfate can reduce hearing loss caused by cisplatin chemotherapy in children with medulloblastoma, a type of brain cancer. It also explores whether low-risk patients can be treated with less intense radiation to reduce side effects. About 225 c…

This Phase 2 trial is testing a vaccine called PEP-CMV in 120 children and young adults with high-grade glioma, diffuse intrinsic pontine glioma (DIPG), or recurrent medulloblastoma. The vaccine targets a common virus (CMV) found in many brain tumors, aiming to boost the immune s…

This study tests a thyroid hormone called liothyronine (L-T3) alone or with standard chemotherapy in children and young adults whose medulloblastoma has come back or worsened. The goal is to see if adding L-T3 can help control the cancer better. About 69 participants will take pa…

This study tests a drug called DFMO (eflornithine) as a maintenance therapy to prevent medulloblastoma, a type of brain cancer, from returning in children. It includes 118 participants aged 0-21 with high-risk or relapsed forms of the disease. The goal is to see if DFMO can exten…

This study tests a drug called PLX038 in adults with brain or spinal cord tumors, including those with changes in the MYC or MYCN genes. The trial has two phases: first, finding the right dose, then checking if it works. Participants receive PLX038 intravenously every three weeks…

This early-phase trial is testing whether a combination of immune-based treatments is safe and feasible for children and young adults (ages 4–30) with a recurrent form of medulloblastoma, a type of brain cancer. The treatment includes personalized dendritic cell vaccines, immune …

This early-phase trial is testing whether adding the drug atovaquone to standard radiation therapy is safe and tolerable for children with certain malignant brain tumors, including high-grade glioma, diffuse midline glioma, and medulloblastoma. The study includes two groups: chil…

This early-phase trial tests a new type of immune therapy for people with certain aggressive brain tumors, including DIPG and high-grade glioma. The treatment uses the patient's own T cells, which are modified in the lab to recognize a protein called GD2 on cancer cells and to pr…

This study is testing a drug called Lutathera in children and young adults (ages 4 to 39) whose brain tumors have come back or are growing despite treatment. The drug delivers radiation directly to tumor cells that show up on a special PET scan. The goal is to find the safest dos…

This early-phase study tests whether adding chemotherapy directly into the spinal fluid (intrathecal topotecan) followed by maintenance pills is feasible for children under 6 with high-risk embryonal brain tumors. About 15 children will receive this treatment after standard high-…

This phase 1 trial tests a drug called 177Lu-DTPA-omburtamab, which delivers radiation directly to tumor cells in children and teens with recurrent or refractory medulloblastoma or other central nervous system (CNS) tumors that have spread. The drug targets a protein called B7-H3…

This early-phase trial tests a personalized RNA vaccine made from each patient's own tumor. It aims to teach the immune system to attack the cancer. The study includes adults with glioblastoma and children with high-grade gliomas or medulloblastoma. The main goals are to see if t…

This early-stage trial tests whether personalized immune cells (T-cells) can safely treat young children with aggressive brain tumors. The T-cells are designed to attack unique markers found on each child's tumor. Up to 12 children under 5 with embryonal brain tumors or recurrent…

This study is testing a combination of two drugs—a targeted radioactive drug (177Lu-DOTATATE) and a PARP inhibitor (olaparib)—in children and teens aged 3 to 18 with certain solid tumors that have come back or not responded to treatment. The goal is to see if the combination is s…

This study tests a new drug called Becotatug Vedotin in children aged 2 to 18 with certain solid tumors that have come back or spread. The drug targets a protein called EGFR found on many cancer cells. The main goal is to see if the drug is safe and to find the best dose, while a…

This early-phase trial is testing a combination of three treatments for children whose brain tumors have come back or not responded to standard care. The experimental drug SGT-53 delivers a normal TP53 gene to tumor cells, and is given alongside the immunotherapy Nivolumab and ra…

This early-phase study tests whether a new type of immunotherapy, called B7-H3 CAR T cells, can be safely given directly into the brain or spinal fluid of children and young adults with aggressive brain tumors like DIPG, DMG, or other recurrent central nervous system cancers. The…

This expanded access program provides the drug DFMO to children and young adults up to age 30 with rare relapsed tumors such as neuroblastoma or medulloblastoma. The goal is to control the disease by targeting specific genetic markers. Participants take DFMO by mouth twice daily,…

This early-phase trial is testing whether adding the drug ibrutinib to a chemo-immunotherapy regimen can safely help children and young adults (ages 6–25) with brain cancers that have come back or not responded to standard treatment. The study combines ibrutinib with indoximod an…

This early-stage study tests an oral drug called ONC206 in adults with brain tumors that have come back. The main goal is to find the safest dose and check for side effects. About 102 participants will take the drug once or several times a week.

This study tests a new radioactive imaging agent called 68Ga-HA-DOTATATE to see if it can safely and accurately detect neuroendocrine tumors using PET/CT scans. About 600 people with known or suspected tumors will receive the agent and be followed for one year. The goal is to imp…

This study tests whether a special PET/MRI scan using a radioactive tracer called Ga68-DOTATATE can better detect certain brain tumors (like meningioma) and tell them apart from changes caused by past treatments. About 200 adults with these tumors will get the scan alongside stan…

This Phase 3 trial tests whether metformin, a widely used diabetes drug, can improve memory and thinking in children and teens who have finished treatment for a brain tumor. About 140 participants aged 7 to 21 will take metformin or a placebo for 16 weeks. Researchers will measur…

This study tests whether children and teens who finished treatment for medulloblastoma can do exercise sessions through video calls. Fifteen participants will take part in personalized workouts three times a week for 12 weeks. The goal is to see if this approach is practical, saf…

Medulloblastoma is a rare but serious childhood brain tumor. Standard treatments can harm the developing brain, and some tumors stop responding to therapy. This study tests whether CDK4/6 inhibitors, drugs already used for breast cancer, can slow or stop medulloblastoma cells gro…

This study is building a biobank of blood, tissue, and health information from 300 children and adults with relapsed or hard-to-treat sonic hedgehog medulloblastoma, a type of brain tumor. Researchers want to learn more about a specific genetic mutation called U1, which may affec…

This study is testing a new approach for children and young adults whose medulloblastoma or ependymoma has come back. Researchers will analyze each person's tumor using drug screening and genetic testing to create a personalized treatment plan. The goal is to find the most effect…

This study follows 184 people who survived medulloblastoma (a type of brain tumor) after receiving radiation therapy. Researchers want to learn about long-term health problems like memory issues, hearing loss, heart and hormone problems, and physical fitness. The goal is to find …

This pilot study is testing whether a combined PET-MRI scan can better detect if a child's brain tumor has returned after treatment. The study will include 5 children with high-grade gliomas or medulloblastomas who have unclear results from standard MRI. The main goal is to see i…

This study looks at how radiation to the brain affects memory in children who had a brain tumor. Researchers will test memory and thinking skills in 130 children ages 4 to 12, starting 2 years after radiation and again 2 years later. The goal is to learn which parts of the brain …

This international registry collects medical data and biological samples from children and adults with rare brain tumors like medulloblastoma and ependymoma. By gathering information from patients not in clinical trials, researchers hope to improve treatment recommendations and i…