A Phase II Study of Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent/Progressive Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors

What this Trial Is About

This research aims to find new treatment options for children with recurrent or progressive medulloblastoma, ependymoma, atypical teratoid rhabdoid tumor (ATRT), and various other central nervous system (CNS) tumors. These tumors have a very poor prognosis despite conventional chemotherapy, high-dose chemotherapy with stem cell rescue, and irradiation. The study evaluates the use of antiangiogenesis therapy, which involves frequent low doses of chemotherapy to target tumor blood vessels while reducing side effects. The study is conducted in separate groups (strata) based on tumor type and treatment history, assessing response rates and other outcomes. Participants receive combinations of drugs including bevacizumab, thalidomide, celecoxib, fenofibrate, etoposide, cyclophosphamide, temozolomide, irinotecan, and intrathecal chemotherapy agents. The treatments are given over one year, following schedules such as oral daily dosing, intravenous infusions biweekly, and intrathecal administrations every few weeks. The study includes both single-arm and randomized two-arm phase 2 designs depending on the tumor group, comparing different drug combinations to assess effectiveness. During the study, participants undergo regular assessments of tumor response, survival rates, toxicity, quality of life, and other markers over a period of up to eight years. The study tracks progression-free survival, overall survival, performance status, and angiogenic factors. Safety monitoring and feasibility are also evaluated. Participants must have normal organ and bone marrow function and meet performance status criteria to be eligible. The total involvement can span several years due to long-term follow-up and monitoring.

Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors