eligibility_summary
Eligible: ≥1y high-risk neuroblastoma (relapsed/refractory/persistent) or ≥6mo metastatic retinoblastoma (IVa extra-CNS or IVb CNS) with evaluable disease, progression or biopsy-proven viable tumor. PS≥60, adequate renal/hepatic, pulmonary (SpO2≥92%) and cardiac function. Exclude: HBV/HCV/HIV, infection/immunodeficiency, DMSO allergy, systemic steroids at collection/infusion, cardiac disease, live vaccine <30d, pregnant/nursing, life expectancy <6mo, NB: active CNS mets, RB: cohort rules.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Phase 1, first-in-human, single-center dose-escalation/expansion study testing GPC2 CAR T cells (autologous, gene-modified cellular immunotherapy, biological) in relapsed/refractory high-risk neuroblastoma and metastatic retinoblastoma. Intervention: patient T cells engineered to express a chimeric antigen receptor that binds glypican-2 (GPC2), a cell-surface heparan sulfate proteoglycan highly expressed on these tumors and enriched in tumor stem-like compartments, with minimal normal tissue expression. Mechanism: CAR engagement of GPC2 triggers T-cell activation, cytokine release, and cytotoxic killing. Targets: GPC2-positive neuroblastoma/retinoblastoma cells (including cancer stem cells) via CAR-driven TCR/CD3ζ and costimulatory signaling pathways.