NCT03638167

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

Completed Phase 1 Last updated 17 December 2025

What this trial tests

Phase 1 trial testing EGFR806-specific chimeric antigen receptor (CAR) T cell in Central Nervous System Tumor, Pediatric in 11 participants. Completed in 22 December 2023.

Timeline

19 March 2019

Primary endpoint
22 December 2023

22 December 2023

Quick facts

Lead sponsor	Seattle Children's Hospital
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	11
Start date	19 March 2019
Primary completion	22 December 2023
Estimated completion	22 December 2023
Sites	1 location across United States

Drugs / interventions tested

• EGFR806-specific chimeric antigen receptor (CAR) T cell — full drug profile →

Conditions studied

• Central Nervous System Tumor, Pediatric — all drugs for Central Nervous System Tumor, Pediatric →
• Glioma — all drugs for Glioma →
• Ependymoma — all drugs for Ependymoma →
• Medulloblastoma — all drugs for Medulloblastoma →

Sponsor

Seattle Children's Hospital

Who can join

Adults 1 to 26, any sex, with Central Nervous System Tumor, Pediatric or Glioma. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. CAR T Cells for Solid Tumors: New Strategies for Finding, Infiltrating, and Surviving in the Tumor Microenvironment.
   Martinez M, Moon EK. · · 2019 · cited 653× · PMID 30804938 · DOI 10.3389/fimmu.2019.00128
2. Brain immunology and immunotherapy in brain tumours.
   Sampson JH, Gunn MD, Fecci PE, Ashley DM. · · 2020 · cited 525× · PMID 31806885 · DOI 10.1038/s41568-019-0224-7
3. Engineered T Cell Therapy for Cancer in the Clinic.
   Zhao L, Cao YJ. · · 2019 · cited 275× · PMID 31681259 · DOI 10.3389/fimmu.2019.02250
4. Locoregionally administered B7-H3-targeted CAR T cells for treatment of atypical teratoid/rhabdoid tumors.
   Theruvath J, Sotillo E, Mount CW, Graef CM, et al · · 2020 · cited 247× · PMID 32341579 · DOI 10.1038/s41591-020-0821-8
5. Intraventricular B7-H3 CAR T Cells for Diffuse Intrinsic Pontine Glioma: Preliminary First-in-Human Bioactivity and Safety.
   Vitanza NA, Wilson AL, Huang W, Seidel K, et al · · 2023 · cited 214× · PMID 36259971 · DOI 10.1158/2159-8290.cd-22-0750
6. CAR-T Therapies in Solid Tumors: Opportunities and Challenges.
   Guzman G, Reed MR, Bielamowicz K, Koss B, et al · · 2023 · cited 121× · PMID 36853475 · DOI 10.1007/s11912-023-01380-x
7. Assessing the Future of Solid Tumor Immunotherapy.
   Guha P, Heatherton KR, O'Connell KP, Alexander IS, et al · · 2022 · cited 85× · PMID 35327456 · DOI 10.3390/biomedicines10030655
8. Immune Checkpoints and CAR-T Cells: The Pioneers in Future Cancer Therapies?
   Hosseinkhani N, Derakhshani A, Kooshkaki O, Abdoli Shadbad M, et al · · 2020 · cited 83× · PMID 33167514 · DOI 10.3390/ijms21218305