Autologous CAR T Cells Targeting GPC3 (RPCAR01) for the Treatment of Advanced or Metastatic GPC3 Expressing Hepatocellular Carcinoma

Summary

This phase I trial studies the side effects and best dose of RPCAR01 chimeric antigen receptor (CAR) T cells and to see how well it works in treating patients with GPC3 expressing hepatocellular carcinoma (HCC) that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or that has spread from where it first started (primary site) to other places in the body (metastatic). In GPC3 expressing HCC cancerous cell tissue overexpresses, or makes too much of, a protein called "GPC3" on the surface of those cells (while only rarely expressed in healthy tissue). RPCAR01 is a genetically modified T cell (a part of the immune system) product that targets GPC3 and decreases the inhibition of T cells by a protein called transforming growth factor beta (TGFB). The drug is prepared by taking T cells from the blood by a procedure called "leukapheresis." The T cells are then modified to make them target GPC3 and disrupt TGFB which may help the body's immune system identify and kill GPC3 tumor cells. Lymphodepletion chemotherapy with cyclophosphamide and fludarabine involves receiving a short course of chemotherapy to kill T cells before receiving the RPCAR01 CAR T cell infusion. Giving RCAR01 CAR T cells may be safe, tolerable, and/or effective in treating patients with advanced or metastatic GPC3 expressing HCC.

Conditions

• Advanced Hepatocellular Carcinoma
• Metastatic Hepatocellular Carcinoma
• Recurrent Hepatocellular Carcinoma
• Refractory Hepatocellular Carcinoma
• Stage III Hepatocellular Carcinoma AJCC v8
• Stage IV Hepatocellular Carcinoma AJCC v8

Interventions

BIOLOGICAL

Anti-GPC3-CAR Autologous T Lymphocytes

Given IV

PROCEDURE

Biospecimen Collection

Undergo blood and tissue sample collection

PROCEDURE

Computed Tomography

Undergo CT

DRUG

Cyclophosphamide

Given IV

PROCEDURE

Echocardiography Test

Undergo ECHO

DRUG

Fludarabine

Given IV

PROCEDURE

Leukapheresis

Undergo leukapheresis

PROCEDURE

Magnetic Resonance Imaging

Undergo MRI

PROCEDURE

Multigated Acquisition Scan

Undergo MUGA

Sponsors & Collaborators

• Roswell Park Cancer Institute

  lead OTHER

Principal Investigators

• Anuradha Krishnamurthy · Roswell Park Cancer Institute

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2026-05-01

Primary Completion

2028-05-30

Completion

2029-05-30

FDA Drug

Yes

Countries

• United States

Study Locations