NCT02442297: iCAR

Phase I Study of Intracranial Injection of T Cells Expressing HER2-specific Chimeric Antigen Receptors (CAR) in Subjects With HER2-Positive Tumors of the Central Nervous System (iCAR)

Active, enrolled Phase 1 Last updated 29 August 2025

What this trial tests

Phase 1 trial testing HER2-specific T cells in Brain Tumor, Recurrent in 10 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

1 April 2016

Primary endpoint
27 April 2022

1 April 2037

Quick facts

Lead sponsor	Baylor College of Medicine
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	1 April 2016
Primary completion	27 April 2022
Estimated completion	1 April 2037
Sites	2 locations across United States

Drugs / interventions tested

• HER2-specific T cells — full drug profile →

Conditions studied

• Brain Tumor, Recurrent — all drugs for Brain Tumor, Recurrent →
• Brain Tumor, Refractory — all drugs for Brain Tumor, Refractory →

Sponsor

Baylor College of Medicine

Who can join

3 and older, any sex, with Brain Tumor, Recurrent or Brain Tumor, Refractory. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Number of patients with dose limiting toxicity after administration of autologous T cells expressing transgenic chimeric antigen receptors (CAR) targeting the HER2 molecule
  Time frame: 6 weeks
  To evaluate the safety of autologous T cells expressing transgenic chimeric antigen receptors (CAR) targeting the HER2 molecule administered into the tumor, tumor resection cavity, and/or cerebrospinal fluid (CSF) space of subjects with progressive recurrent or refractory HER2-positive primary central nervous system (CNS) tumor or HER2 positive tumor metastatic to the CNS, excluding diffuse intrap

Sponsor's own description

This study is for patients that have brain cancer. The body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells. Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting immune cells present in the blood that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise, but have not been strong enough to cure most patients.

The antibody used in this study is called anti-HER2 (Human Epidermal Growth Factor Receptor 2). This antibody sticks to tumor cells because of a substance on the outside of these cells called HER2. Many types of brain tumors are positive for HER2 . HER2 antibodies have been used to treat people with HER2-positive cancers. For this study, the HER2 antibody has been changed so that instead of floating free in the blood it is now attached to T cells. When an antibody is joined to a T cell in this way it is called a chimeric antigen receptor (CAR). These CAR-T cells seem to be able to kill tumors like the one these patients have, but they don't last very long and so their chances of fighting the cancer are limited. Therefore, developing ways to prolong the life of these T cells should help them fight cancer.

These HER2-CAR T cells are an investigational product not approved by the Food and Drug Administration.

The purpose of this study is to find the largest safe dose of HER2-CAR T cells, to learn what the side effects are, and to see whether this experimental intervention might help patients with brain tumors who volunteer to test this new agent.

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. Driving CAR T-cells forward.
   Jackson HJ, Rafiq S, Brentjens RJ. · · 2016 · cited 457× · PMID 27000958 · DOI 10.1038/nrclinonc.2016.36
4. Glioblastoma targeted therapy: updated approaches from recent biological insights.
   Touat M, Idbaih A, Sanson M, Ligon KL. · · 2017 · cited 308× · PMID 28863449 · DOI 10.1093/annonc/mdx106
5. CAR T Cell Therapy for Solid Tumors: Bright Future or Dark Reality?
   Wagner J, Wickman E, DeRenzo C, Gottschalk S. · · 2020 · cited 284× · PMID 32979309 · DOI 10.1016/j.ymthe.2020.09.015
6. Engineered T Cell Therapy for Cancer in the Clinic.
   Zhao L, Cao YJ. · · 2019 · cited 275× · PMID 31681259 · DOI 10.3389/fimmu.2019.02250
7. 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
8. Understanding the immunosuppressive microenvironment of glioma: mechanistic insights and clinical perspectives.
   Lin H, Liu C, Hu A, Zhang D, et al · · 2024 · cited 232× · PMID 38720342 · DOI 10.1186/s13045-024-01544-7