NCT07087002

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

Phase I Clinical Trial of GPC2 Chimeric Antigen Receptor T (GPC2-CAR T) Cells for Relapsed or Refractory Medulloblastoma in Children and Young Adults

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: Stanford University · Academic / Other
• Sex: All
• Age: 1 Year – 30 Years
• Healthy volunteers: Not accepted

Summary

This is a single-site, open-label Phase 1 clinical trial evaluating the feasibility, safety, and preliminary activity of autologous GPC2-targeted chimeric antigen receptor (CAR) T cells administered via intracerebroventricular (ICV) infusion in children and young adults with relapsed or refractory medulloblastoma or other eligible Central Nervous System (CNS) embryonal tumors.

Conditions

• Medulloblastoma
• Central Nervous System Embryonal Tumor
• Refractory Medulloblastoma
• Recurrent Medulloblastoma
• Pediatric Brain Tumor
• Embryonal Tumor With Multilayered Rosettes (ETMR)
• Pineoblastoma
• Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS
• CNS Neuroblastoma
• FOXR2-activated

Interventions

Type	Name	Description
BIOLOGICAL	GPC2-CAR T cells	Autologous T cells transduced with retroviral vector encoding a second-generation GPC2-targeted chimeric antigen receptor (GPC2-CAR), administered intracerebroventricularly. Up to 8 doses are given every 28 days, following an intrapatient dose escalation schema.
DRUG	Fludarabine	Administered as part of a lymphodepleting chemotherapy regimen prior to GPC2-CAR T cell infusion. Dose: 30 mg/m²/day for 3 days.
DRUG	Cyclophosphamide	Administered with fludarabine for lymphodepletion. Dose: 500 mg/m²/day for 3 days.

Timeline

• Start date: 2025-08-28
• Primary completion: 2027-08-01
• Completion: 2027-08-01
• First posted: 2025-07-25
• Last updated: 2026-01-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study