Phase 1 Study of Personalized T Cell Immunotherapy for Young Children With Embryonal Brain Tumors and Patients Up to 30 Years With Recurrent Ependymoma

What this Trial Is About

This trial investigates the safety and feasibility of a new immunotherapy using multi-tumor antigen specific cytotoxic T lymphocytes TSA-T tailored to individual tumor-specific antigens found in pediatric brain tumors. It focuses on young patients with embryonal central nervous system CNS malignancies who often cannot receive irradiation and instead undergo intensive chemotherapy and stem cell rescue, yet still face high relapse rates. The study aims to assess this personalized immunotherapy after standard treatments in children under 5 years old with embryonal brain tumors and in children, adolescents, and young adults with recurrent ependymoma. Participants are divided into two groups receiving different backbone therapies Group A includes children under 5 years with newly diagnosed embryonal brain tumors treated with surgery and chemotherapy cycles, possibly including radiation Group B includes patients aged 1 to under 30 years with recurrent ependymoma receiving surgical re-resection, re-irradiation, and optional bridging chemotherapy. After these treatments, patients receive TSA-T infusions via a Rickham reservoir, starting at a low dose and escalating according to safety rules, with up to 8 infusions spaced several weeks apart. The study will determine the maximum tolerated dose and recommended phase 2 dose based on safety data from both groups combined. Throughout the study, participants undergo assessments including clinical, immunological, and biomarker studies to monitor safety, tumor response, and immune effects. Safety is evaluated primarily by dose-limiting toxicities in the first 42 days after infusion, while tumor responses and survival are tracked up to 5 years. Patients must adhere to the treatment schedule and have specific medical criteria met before each infusion. The study involves tissue collection, imaging, laboratory tests, and neurological evaluations to measure outcomes and monitor the effects of the therapy during and after treatment.

Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)