Phase 1 Study of GPC2-Targeted CAR T Cell Therapy for Children with Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma

What this Trial Is About

This research aims to evaluate the safety and feasibility of using GPC2-directed CAR T cells in patients with advanced neuroblastoma or retinoblastoma who have relapsed or refractory disease. These children face poor outcomes despite existing intense treatments, and GPC2 is a promising target due to its high expression on tumor cells but low presence on normal tissues. The trial is a first-in-human, open-label, phase 1 dose escalation and expansion study designed to assess safety, tolerability, and manufacturing feasibility of this novel therapy. Participants will receive genetically modified autologous T cells engineered to target GPC2. The study includes two arms a dose escalation arm to determine the maximum tolerated dose using a standard 33 design, followed by a dose expansion arm to further assess safety and preliminary effectiveness at a safe dose. The treatment is given once the dose is established, and further patients may be enrolled to evaluate response rates and safety profiles. During the study, participants will be closely monitored for adverse events and treatment response over five years. Researchers will assess safety outcomes such as maximum tolerated dose and frequency of adverse events, and secondary outcomes including cell manufacturing feasibility, persistence of CAR T cells, and preliminary clinical activity. Participants will undergo regular evaluations including imaging, laboratory tests, and clinical assessments to track disease status and treatment effects throughout the study and follow-up period.

GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma