Atypical teratoid rhabdoid tumor

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 two oral drugs, eflornithine (DFMO) and AMXT 1501, in children and young adults up to age 26 with certain cancers that have come back or not responded to treatment. The cancers include neuroblastoma, brain tumors, and sarcomas. The goal is to find a safe dose and…

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 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 study tests a combination of two drugs, gemcitabine and paxalisib, in children, teens, and young adults whose atypical teratoid rhabdoid tumor (ATRT) has come back or gotten worse. The goal is to find a safe dose and see if the drugs can control the tumor. About 29 participa…

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 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-stage trial tests whether a device called SonoCloud-9 can safely open the blood-brain barrier in children aged 5 to 17 with recurrent malignant brain tumors. The barrier normally blocks chemotherapy from reaching the brain. By using low-intensity ultrasound just before…

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 study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…