Phase 1 Study of GPC2-Targeted CAR T Cell Therapy Delivered into the Brain Ventricles for Children and Young Adults with Relapsed or Refractory Medulloblastoma and Other CNS Embryonal Tumors

What this Trial Is About

Researchers are evaluating a new approach using GPC2-targeted chimeric antigen receptor CAR T cells to treat children and young adults with relapsed or refractory medulloblastoma and other similar central nervous system embryonal tumors. This Phase 1 clinical trial focuses on the safety, feasibility, and initial effects of this treatment delivered directly into the brains ventricles. The study is led by Stanford University and includes participants aged 1 to 30 years with confirmed diagnoses and specific tumor markers. Participants will first undergo leukapheresis to collect their immune cells, which are then modified to produce GPC2-CAR T cells. Before receiving these cells, patients undergo lymphodepleting chemotherapy with fludarabine and cyclophosphamide. Afterward, they receive up to eight intracerebroventricular infusions of the modified cells every 28 days, with doses gradually increased within each patient. This treatment schedule follows a set dose escalation strategy to monitor patient response and safety. During the trial, participants will be closely monitored for manufacturing success of the CAR T cells and any dose-limiting toxicities within the first treatment cycle. Additional assessments include imaging and cerebrospinal fluid tests to evaluate disease status. Researchers will also track objective response rates, progression-free survival, and overall survival for up to two years after infusion. Safety and organ function tests, performance status evaluations, and pregnancy testing are part of ongoing monitoring throughout the study period.

GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults