Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy for High-Risk Embryonal Brain Tumors in Children Under 6 Years After High-Dose Chemotherapy

What this Trial Is About

This research aims to evaluate the feasibility of adding intrathecal chemotherapy and maintenance therapy after high-dose chemotherapy for treating newly diagnosed high-risk embryonal brain tumors HR-EBTs in children under 6 years of age. The trial focuses on children with specific central nervous system tumors confirmed by histologic or molecular diagnosis, including atypical teratoid rhabdoid tumor, various medulloblastoma groups, pineoblastoma, neuroblastoma, and other related embryonal tumors. The study also explores genetic characteristics of these tumors through advanced genomic analyses. Participants will undergo a comprehensive treatment plan starting with three cycles of induction chemotherapy using multiple agents alongside intrathecal cytarabine and hydrocortisone. This is followed by three cycles of consolidation chemotherapy with carboplatin, thiotepa, and autologous stem cell rescue. After consolidation, patients receive risk-adapted maintenance chemotherapy for 48 to 54 weeks, depending on their risk group. Maintenance includes monthly intrathecal topotecan with either tamoxifen and retinoic acid for low-risk patients or a combination of isotretinoin, celecoxib, etoposide, temozolomide, and cyclophosphamide for high-risk patients. Throughout the study, participants will have baseline and follow-up brain and spine MRIs and other assessments to monitor response using specialized pediatric neuro-oncology criteria. Follow-up visits occur every three months for two years after treatment to evaluate progression-free and overall survival. Safety and treatment-related toxicities are closely monitored. The total study duration per participant is about two years, including treatment and follow-up, with additional genomic studies to understand tumor biology.

Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation