Phase I Trial of RNA-Lipid Particle Vaccines for Children and Young Adults with Recurrent or Progressive Medulloblastoma Evaluating Safety and Dose

What this Trial Is About

Researchers are studying RNA-lipid particle RNA-LP vaccines in children and young adults with recurrent or progressive Medulloblastoma, a type of brain tumor. This first-in-human Phase I trial aims to evaluate how feasible it is to manufacture these vaccines, determine their safety, and find the highest dose patients can tolerate. The study includes a dose-escalation approach to identify the maximum tolerated dose. The trial has three parts surgery to obtain tumor material, salvage therapy involving radiation andor chemotherapy, and immunotherapy with RNA-LP vaccines. After surgery and salvage therapy, participants will receive three RNA-LP vaccine doses every two weeks, followed by 12 monthly booster doses, totaling 15 vaccines over about 14 months. Tumor samples are collected and sent to the University of Florida for processing. Participants will be monitored throughout the study and followed until death from any cause. Researchers will conduct MRI scans and clinical evaluations every three months during the first year after immunotherapy to check disease progression. Safety and manufacturing feasibility will be assessed up to 15 weeks, and the maximum tolerated dose will be evaluated over up to five years after surgery.

RNA-lipid Particle (RNA-LP) Vaccines for Recurrent/Progressive Medulloblastoma (MB)