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Molecular and Clinical Risk Directed Therapy for Infants and Young Children with Newly Diagnosed Medulloblastoma

Trial Status: active

This phase II trial tests the effect of using tumor molecular groups and subgroups to guide treatment in infants and young children with newly diagnosed medulloblastoma. Medulloblastoma in children is a fast-growing tumor. This tumor can spread into the fluid that surrounds the brain and spinal cord (cerebrospinal fluid or CSF). In older children, surgery followed by high doses of radiation therapy and chemotherapy are usually given to the brain and spine to prevent the tumor from spreading. However, radiation therapy to the developing brain and spine of infants and young children may cause permanent problems with thinking, learning, and growing. In these infants and young children, researchers would like to postpone or avoid using radiation therapy if possible. Anti-cancer drugs (chemotherapy) have been used to kill brain tumor cells in patients. Chemotherapy allows the brain more time to develop before the radiation is given, or, in some cases, it may prevent radiation from being needed at all for treatment. Research studies from the last twenty years have shown that a fraction of medulloblastoma cases may be cured by just chemotherapy, allowing avoidance of radiation, and thus protecting developing brains from problems with thinking and learning. In this study researchers will use molecular groups and subgroups of medulloblastoma to differentiate between medulloblastoma patients who are more likely to respond to just chemotherapy from those who need radiation. In this study, a new method for diagnosing specific molecular groups and subgroups of medulloblastoma based on biological features of the tumor will be used. The method is called “methylation”, which detects molecular patterns (marks) on deoxyribonucleic acid (DNA) of the cells from the tumor. Different groups and subgroups of medulloblastoma will be identified based on their unique molecular patterns. Treatment will then be guided based on the tumor group and subgroup. The main goal of this study is to determine whether using tumor molecular group and subgroup to guide treatment intensity results in improved treatment efficacy while limiting long-term treatment-related problems in infants and young children.