CAR T-Cell Therapy for Liver Cancer
(GAP Trial)

Recruiting in Palo Alto (17 mi)

Overseen byAndras A Heczey, M.D.

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Baylor College of Medicine

No Placebo Group

Trial Summary

What is the purpose of this trial?

This study enrolls patients who have GPC3-positive solid tumors currently. Patients may be considered if the cancer has come back, has not gone away after standard treatment or the patient cannot receive standard treatment. This research study uses special immune system cells called GAP T cells, a new experimental treatment. 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 blood cells 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. Investigators have found from previous research that they can put a new gene into T cells that will make them recognize cancer cells and kill them. In preclinical studies, the investigators made several genes called a chimeric antigen receptor (CAR), from an antibody called GC33 that recognizes glypican-3, a proteoglycan found on solid tumors including pediatric liver cancers (GPC3-CAR). This study will test T cells genetically engineered with a GPC3-CAR (GAP T cells) in patients with GPC3-positive solid tumors (currently only enrolling liver tumors). The GAP T cells are an investigational product not approved by the Food and Drug Administration. The purpose of this study is to find the biggest dose of GAP T cells that is safe, to see how long they last in the body, to learn what the side effects are and to see if the GAP T cells will help people with GPC3-positive solid tumors. This study enrolls patients who have GPC3-positive solid tumors (currently only enrolling liver tumors).

Research Team

Andras A Heczey, M.D.

Principal Investigator

Baylor College of Medicine

Eligibility Criteria

This trial is for children and young adults aged 1 to 21 with GPC3-positive solid liver tumors that have either returned after treatment, haven't responded to standard treatments, or can't be treated conventionally. Participants need a reasonable life expectancy and good performance status, but those with organ transplants, HIV, active infections (except certain hepatitis), severe reactions to Cytoxan or Fludara, or allergies to murine proteins are excluded.

Inclusion Criteria

I can do most activities but may need help.

I am mostly able to carry out daily activities.

Adequate organ function: Creatinine clearance as estimated by Cockcroft Gault or Schwartz ≥ 60 ml/min, serum AST< 5 times ULN, total bilirubin < 3 times ULN for age, INR ≤1.7 (for patients with hepatocellular carcinoma only), absolute neutrophil count > 500/microliter, platelet count > 25,000/microliter (can be transfused), Hgb ≥7.0 g/dl (can be transfused), pulse oximetry >90% on room air, Recovered from acute toxic effects of all prior chemotherapy and investigational agents before entering this study, Sexually active patients must be willing to utilize one of the more effective birth control methods for 3 months after the T-cell infusion, Informed consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent

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Exclusion Criteria

I am allergic to mouse protein products or have human anti-mouse antibodies.

I have had an organ transplant.

I am not on high doses of steroids or can stop them 24 hours before CAR T cell therapy.

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Treatment Details

Interventions

Cytoxan (Alkylating agents)
Fludara (Anti-metabolites)
GAP T cells (CAR T-cell Therapy)

Trial OverviewThe GAP clinical trial is testing a new therapy using GAP T cells—immune cells modified with a chimeric antigen receptor targeting glypican-3 on cancer cells. The study aims to determine the highest safe dose of these engineered T cells, their lifespan in the body, side effects they may cause, and their effectiveness against liver tumors.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: GAP T cells + Fludarabine and CytoxanExperimental Treatment3 Interventions

GPC3-Car (GAP T cells) along with lymphodepleting chemotherapy (Cytoxan and Fludarabine) will be administered to patients with GPC3-positive solid tumors.