Study of Induction Chemotherapy, High-Dose Chemotherapy, and Radiation Therapy for Children with Newly Diagnosed Embryonal Tumor With Multilayered Rosettes ETMR

What this Trial Is About

The trial evaluates treatments for children with newly diagnosed Embryonal Tumor With Multilayered Rosettes ETMR, focusing on induction chemotherapy, high-dose chemotherapy, and focal radiation therapy. This phase 2 study aims to assess progression-free survival and overall survival in participants with different extents of tumor resection and metastatic status. The research also explores biomarkers in blood and cerebrospinal fluid and aims to better understand the tumors genetic makeup. Participants first undergo surgical removal of the tumor, either gross total or sub-total resection, before joining one of four cohorts based on tumor status and metastasis. Cohorts 1 and 2 include participants with gross-total resection and no metastasis, receiving either induction chemotherapy with early radiotherapy or high-dose chemotherapy with stem cell rescue. Cohorts 3A and 3B include participants with metastatic or residual disease, treated similarly but with longer follow-up. Treatments last between 12 to 24 weeks depending on the cohort, with options for radiotherapy and chemotherapy combinations. During the study, participants are monitored for up to two years for cohorts 1 and 2, and up to five years for cohorts 3A and 3B. Assessments include survival outcomes and biomarker analysis from tumor tissue, blood, and cerebrospinal fluid samples. Participants must meet specific health and organ function criteria and are followed under a pediatric neuro-oncology protocol until death or withdrawal. The primary outcome is progression-free survival at six months for cohort 1, with secondary outcomes including two-year progression-free survival and overall survival across cohorts.

Embryonal Tumor With Multilayered Rosettes