Personalized Treatment Using Real-Time Drug Screening and Genomic Testing for Children and Young Adults with Relapsed Medulloblastoma and Ependymoma

What this Trial Is About

Researchers are evaluating a personalized treatment approach for children and young adults with relapsed medulloblastoma or ependymoma. The study is a multi-center pilot and efficacy trial aiming to test the feasibility of using real-time drug screening and genomic sequencing of tumor tissue to guide individualized treatment plans. This approach is designed to offer the most effective and least toxic therapies based on each patients tumor molecular characteristics and to improve future understanding and treatment of these tumors. Participants will receive individualized treatment recommendations including up to four FDA-approved drugs based on results from high-throughput drug screening, whole exome sequencing, and RNA sequencing performed on tissue obtained during surgical resection or biopsy. The specialized tumor board will provide treatment plans within 21 business days of tissue collection. Participants may continue treatment for up to two years or until disease progression, with the possibility to extend therapy if clinical benefits and disease control are observed. During the study, participants undergo surgery to obtain tumor tissue, followed by molecular testing and drug screening. Researchers will collect blood samples and biospecimens over time to explore biomarkers related to tumor burden and therapy response. Participants will be monitored for treatment safety, tumor progression, quality of life, and overall survival for up to five years. The primary outcome measures include timely completion of treatment recommendations and progression-free survival, with secondary assessments of adverse events and patient-reported outcomes.

Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma