Phase 1 Study of iC9-GD2-CAR T Cells for Treating Relapsed or Refractory Pediatric and Young Adult Brain Tumors

What this Trial Is About

Researchers are evaluating the safety and feasibility of a new treatment called iC9-GD2-CAR T-cells in children and young adults with relapsed or refractory malignant central nervous system tumors. This Phase I study focuses on patients with different types of brain tumors, including medulloblastoma, high-grade gliomas, and other rare CNS tumors. The treatment includes a safety feature called a suicide gene to help manage possible side effects. The treatment involves collecting the patients own T cells through leukapheresis, then modifying them to target GD2, a marker on tumor cells. Before receiving the modified cells, patients undergo chemotherapy to reduce immune cells temporarily lymphodepleting regimen. Afterwards, patients receive a single intravenous infusion of the engineered CAR T cells at a dose ranging from 1.0 to 6.0 x 106 cells per kg, depending on their tumor type. The study has three groups based on tumor classification and risk, which helps test safety sequentially. Participants will be closely monitored for safety and response, including imaging studies, blood tests, and clinical assessments. They will be followed actively for 5 years to track disease status and treatment effects, with an additional 15-year follow-up to meet gene therapy regulations. Key measures include safety evaluation, CAR T cell persistence and expansion, tumor response, and survival outcomes. The study aims to define the maximum tolerated dose and recommended dose of this investigational therapy.

GD2-CAR T Cells for Pediatric Brain Tumours