A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-RiskWNT-Driven Medulloblastoma Patients
Primary Objective To estimate the progression-free survival (PFS) of children ≥ 3 years of age with WNT-driven average-risk medulloblastoma using reduced craniospinal radiotherapy (CSI) (18 Gy) with a limited target volume boost to the tumor bed of 36 Gy for a total of 54 Gy and reduced chemotherapy approach (no vincristine during radiotherapy and reduced-dose maintenance chemotherapy) and to monitor the PFS for early evidence that the outcome is unacceptable.
Secondary Objectives To prospectively test the hypothesis that DNA methylation profiling will accurately classify WNT-driven medulloblastoma. To prospectively evaluate and longitudinally model the cognitive, social, emotional, behavioral and Quality of Life (QoL) functioning of children who are treated with reduced CSI (18 Gy) with a limited target volume boost to the tumor bed (to a total of 54 Gy) and reduced chemotherapy (reduced cisplatin, vincristine and CCNU).
Exploratory Objectives To explore whether DNA methylation profiling of medulloblastoma samples will result in a predictive classification scheme for the Sonic Hedgehog (SHH), Group 3 and Group 4 medulloblastoma subgroups according to the Heidelberg classifier. To describe the audiologic and endocrinologic toxicities, as well as peripheral neuropathy in children treated with reduced CSI (18 Gy) with a limited target volume boost to the tumor bed (to a total of 54 Gy) and reduced cisplatin and vincristine chemotherapy.