NCT03638167

EGFR806-specific CAR T Cell Locoregional Immunotherapy for EGFR-positive Recurrent or Refractory Pediatric CNS Tumors

Phase 1 Study of EGFR806-specific CAR T Cell Locoregional Immunotherapy for EGFR-positive Recurrent or Refractory Pediatric Central Nervous System Tumors

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Seattle Children's Hospital · Academic / Other
• Sex: All
• Age: 1 Year – 26 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1 study of central nervous system (CNS) locoregional adoptive therapy with autologous CD4+ and CD8+ T cells that are lentivirally transduced to express an EGFR806 specific chimeric antigen receptor (CAR) and EGFRt. CAR T cells are delivered via an indwelling catheter into the tumor cavity or the ventricular system in children and young adults with recurrent or refractory EGFR-positive CNS tumors. The primary objectives of this protocol are to evaluate the feasibility, safety, and tolerability of CNS-delivered fractionated CAR T cell infusions employing intra-patient dose escalation. Subjects with supratentorial tumors will receive sequential EGFR806-specific CAR T cells delivered into the tumor resection cavity, subjects with infratentorial tumors will receive sequential CAR T cells delivered into the fourth ventricle, and subjects with leptomeningeal disease will receive sequential CAR T cells delivered into the lateral ventricle. The secondary objectives are to assess CAR T cell distribution within the cerebrospinal fluid (CSF), the extent to which CAR T cells egress into the peripheral circulation, and EGFR expression at recurrence of initially EGFR-positive tumors. Additionally, tumor response will be evaluated by magnetic resonance imaging (MRI) and CSF cytology. The exploratory objectives are to analyze CSF specimens for biomarkers of anti-tumor CAR T cell presence and functional activity.

Conditions

• Central Nervous System Tumor, Pediatric
• Glioma
• Ependymoma
• Medulloblastoma
• Germ Cell Tumor
• Atypical Teratoid/Rhabdoid Tumor
• Primitive Neuroectodermal Tumor
• Choroid Plexus Carcinoma
• Pineoblastoma

Interventions

Type	Name	Description
BIOLOGICAL	EGFR806-specific chimeric antigen receptor (CAR) T cell	Autologous CD4+ and CD8+ T cells lentivirally transduced to express an EGFR806 specific chimeric antigen receptor (CAR) and EGFRt given via indwelling central nervous system (CNS) catheter

Timeline

• Start date: 2019-03-19
• Primary completion: 2023-12-22
• Completion: 2023-12-22
• First posted: 2018-08-20
• Last updated: 2025-12-17

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study