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

NCT03638167 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2025-12-17

No results posted yet for this study

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

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

Sponsors & Collaborators

  • Seattle Children's Hospital

    lead OTHER

Principal Investigators

  • Juliane Gust, MD, PhD · Seattle Children's Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
26 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-03-19
Primary Completion
2023-12-22
Completion
2023-12-22
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03638167 on ClinicalTrials.gov