Global Study to Improve Diagnosis and Treatment of Children, Teens, and Young Adults With Intracranial Ependymoma Under 22 Years

What this Trial Is About

Researchers are conducting an international clinical program to improve diagnosis, treatment, and understanding of childhood ependymoma in patients under 22 years old. The study aims to harmonize staging and care standards, assess biological markers, and develop treatment recommendations based on age, tumor location, and surgical outcomes. Patients are categorized into three groups based on residual disease and eligibility for radiotherapy to test different therapeutic strategies. The study includes three treatment strata Stratum 1 evaluates the impact of a 16-week chemotherapy regimen with VEC-CDDP after surgery and conformal radiotherapy in patients aged over 12 months to under 22 years with completely removed tumors. Stratum 2 examines the addition of high-dose methotrexate to VEC chemotherapy in patients with residual disease, followed by conformal radiotherapy and possibly an 8 Gy radiotherapy boost for inoperable residual tumors. Stratum 3 studies dose-dense chemotherapy with or without valproate in children under 12 months or those ineligible for radiotherapy. Patients not meeting these criteria enter an observational registry. Participants undergo centralized pathology and imaging reviews to confirm diagnosis and tumor resection status. Assessments include MRI scans, biological marker evaluations, and response to treatments. Researchers monitor progression-free survival, gross total resection rates, treatment response, neuropsychological and neuroendocrine effects, and safety over several years. The study involves scheduled visits, laboratory tests, and follow-up for up to five years to evaluate long-term outcomes.

An International Clinical Program for the Diagnosis and Treatment of Children With Ependymoma