Ignite Creation Date:
2025-12-24 @ 1:12 PM
Ignite Modification Date:
2025-12-27 @ 9:54 PM
Study NCT ID:
NCT05003895
Status:
RECRUITING
Last Update Posted:
2025-05-22
First Post:
2021-08-12
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
GPC3 Targeted CAR-T Cell Therapy in Advanced GPC3 Expressing Hepatocellular Carcinoma (HCC)
Sponsor:
National Cancer Institute (NCI)
Organization:
Study Overview
Official Title:
Phase I Study of GPC3 Targeted CAR-T Cell Therapy in Advanced GPC3 Expressing Hepatocellular Carcinoma (HCC)
Status:
RECRUITING
Status Verified Date:
2025-05-20
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background:
A new cancer treatment takes a person s own T cells, modifies them in a laboratory so they can better fight cancer cells, and then gives them back to the person. Researchers want to see if this treatment can help people with a certain type of liver cancer.
Objective:
To see if a personalized immune treatment, anti-GPC3 CAR-T cells, is safe.
Eligibility:
Adults aged 18 years and older who have Glypican-3 (GPC3) positive HCC, a type of liver cancer.
Design:
Participants will be screened with the following:
Blood and urine tests
Medical history
Physical exam
Heart function tests
Review of their symptoms and their ability to perform their normal activities
Tumor biopsy
Imaging scan of the chest, abdomen, and pelvis
Participants will have leukapheresis. They may have an IV (intravenous catheter, a small tube put into an arm vein) inserted into each arm or get a central line. Blood will be removed. A machine will separate the white blood cells from their blood. The rest of their blood will be returned to them.
Participants will be admitted to the hospital for about 2 weeks. They will get the chemotherapy drugs fludarabine and cyclophosphamide by IV for 3 days. Then they will receive the modified white blood cells by IV.
Participants will have frequent blood draws. They will give blood and tumor samples for research.
Participants will have follow-up visits for the next 15 years. Then they will be contacted by email or phone for the rest of their life. If their disease does not get worse after 5 years, they will continue to be invited to do imaging studies every 6 months.
A new cancer treatment takes a person s own T cells, modifies them in a laboratory so they can better fight cancer cells, and then gives them back to the person. Researchers want to see if this treatment can help people with a certain type of liver cancer.
Objective:
To see if a personalized immune treatment, anti-GPC3 CAR-T cells, is safe.
Eligibility:
Adults aged 18 years and older who have Glypican-3 (GPC3) positive HCC, a type of liver cancer.
Design:
Participants will be screened with the following:
Blood and urine tests
Medical history
Physical exam
Heart function tests
Review of their symptoms and their ability to perform their normal activities
Tumor biopsy
Imaging scan of the chest, abdomen, and pelvis
Participants will have leukapheresis. They may have an IV (intravenous catheter, a small tube put into an arm vein) inserted into each arm or get a central line. Blood will be removed. A machine will separate the white blood cells from their blood. The rest of their blood will be returned to them.
Participants will be admitted to the hospital for about 2 weeks. They will get the chemotherapy drugs fludarabine and cyclophosphamide by IV for 3 days. Then they will receive the modified white blood cells by IV.
Participants will have frequent blood draws. They will give blood and tumor samples for research.
Participants will have follow-up visits for the next 15 years. Then they will be contacted by email or phone for the rest of their life. If their disease does not get worse after 5 years, they will continue to be invited to do imaging studies every 6 months.
Detailed Description:
Background:
Hepatocellular carcinoma (HCC) is the fifth most common cancer worldwide and the second leading cause of cancer-associated mortality with an average life expectancy of 6-9 months
Despite the success of several studies showing efficacy in treating HCC, most clinical trials have failed to prove a survival advantage.
Adoptive T-cell therapy exploits the natural ability of T-cells to recognize and eliminate their target.
GPC3 is a cell surface protein that is expressed in nearly all HCC yet is undetectable in normal adult hepatic tissues.
We want to evaluate the role of GPC3 targeted chimeric antigen receptor (CAR)-T cells in advanced GPC3 expressing HCC.
Objective:
To determine the safety and feasibility of T-cells, expressing a novel humanized anti-GPC3 chimeric antigen receptor, in participants with advanced HCC, expressing GPC3.
Eligibility:
Histologically confirmed diagnosis of hepatocellular carcinoma
GPC3 positivity of \>= 25% by immunohistochemistry
At least 1 measurable lesion by RECIST v 1.1 criteria
Age \>= 18 years
Design:
We plan to conduct a phase I dose escalation designed clinical trial using CAR (hYP7)-T cells in participants with GPC3 expressing advanced hepatocellular carcinoma.
Participants will undergo leukapheresis
Participants will receive a lymphocyte depleting chemotherapy conditioning regimen with the intent of enhancing the activity of the infused CAR-expressing T cells
Following the T cell infusion, there is a mandatory 9-day inpatient hospitalization to monitor for toxicity.
The participants will be closely monitored during the first year after cell infusion and followed for life.
Hepatocellular carcinoma (HCC) is the fifth most common cancer worldwide and the second leading cause of cancer-associated mortality with an average life expectancy of 6-9 months
Despite the success of several studies showing efficacy in treating HCC, most clinical trials have failed to prove a survival advantage.
Adoptive T-cell therapy exploits the natural ability of T-cells to recognize and eliminate their target.
GPC3 is a cell surface protein that is expressed in nearly all HCC yet is undetectable in normal adult hepatic tissues.
We want to evaluate the role of GPC3 targeted chimeric antigen receptor (CAR)-T cells in advanced GPC3 expressing HCC.
Objective:
To determine the safety and feasibility of T-cells, expressing a novel humanized anti-GPC3 chimeric antigen receptor, in participants with advanced HCC, expressing GPC3.
Eligibility:
Histologically confirmed diagnosis of hepatocellular carcinoma
GPC3 positivity of \>= 25% by immunohistochemistry
At least 1 measurable lesion by RECIST v 1.1 criteria
Age \>= 18 years
Design:
We plan to conduct a phase I dose escalation designed clinical trial using CAR (hYP7)-T cells in participants with GPC3 expressing advanced hepatocellular carcinoma.
Participants will undergo leukapheresis
Participants will receive a lymphocyte depleting chemotherapy conditioning regimen with the intent of enhancing the activity of the infused CAR-expressing T cells
Following the T cell infusion, there is a mandatory 9-day inpatient hospitalization to monitor for toxicity.
The participants will be closely monitored during the first year after cell infusion and followed for life.
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 21-C-0030 | None | None | View |