HepatoblastomaPhase 1NCT06198296
Immunotherapy For Adults With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells
A Phase 1 study testing 21.15.GPC3-CAR T cells in people with Hepatoblastoma and Hepatocellular Carcinoma, run by Baylor College of Medicine at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Hepatoblastoma, Hepatocellular Carcinoma, Wilms Tumor
- What is being tested?
- 21.15.GPC3-CAR T cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Jun 2025to 1 Feb 2043
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Procurement Inclusion Criteria:
- Diagnosis of GPC3-positive* solid tumors (as determined by immunohistochemistry with an extent score of >=Grade 2 [>25% positive tumor cells] and an intensity score of >= 2 [scale 0-4]).
- Age ≥21 years
- Lansky or Karnofsky score ≥60%
- Life expectancy ≥16 weeks
- Barcelona Clinic Liver Cancer Stage A, B or C (- Child-Pugh-Turcotte score <7 (for patients with hepatocellular carcinoma only)
- Informed consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent * GPC3 expression will be evaluated by standard immunohistochemistry (IHC) at Texas Patients's Hospital/Baylor College of Medicine, Department of Pathology for all patients to meet procurement eligibility. All patients will send at least 5 unstained slides.
Procurement Exclusion Criteria:
- History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies).
- History of organ transplantation
- Known HIV positivity
- Active bacterial, fungal or viral infection (except Hepatitis B or Hepatitis C virus infections)
Treatment Inclusion Criteria:
- Diagnosis of GPC3-positive solid tumor
- Age ≥ 21 years
- Barcelona Clinic Liver Cancer Stage A, B or C
- Life expectancy of ≥ 12 weeks
- Lansky or Karnofsky score ≥ 60%
- Child-Pugh-Turcotte score < 7
- Adequate organ function:
- Creatinine clearance as estimated by Cockcroft Gault or Schwartz ≥ 60 ml/min
- total bilirubin < 3 times ULN for age
- INR ≤1.7 (for patients with hepatocellular carcinoma only)
- absolute neutrophil count > 500/µl
- platelet count > 25,000/µl (can be transfused)
- Hgb ≥ 7.0 g/dl (can be transfused)
- Pulse oximetry >90% on room air
- Refractory or relapsed disease after treatment with up- front therapy and at least one salvage treatment cycle
- Recovered from acute toxic effects of all prior chemotherapy and investigational agents before entering this study, as determined by history and physical exam
- 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
Treatment Exclusion Criteria:
- Pregnancy or lactation
- Uncontrolled infection
- Systemic steroid treatment (greater than or equal to 0.5 mg prednisone equivalent/kg/day, dose adjustment or discontinuation of medication must occur at least 24hrs prior to CAR T cell infusion)
- Known HIV positivity
- Active bacterial, fungal or viral infection (except Hepatitis B or Hepatitis C virus infections)
- History of organ transplantation
- History of hypersensitivity reactions to murine protein-containing products OR presence of human anti-mouse antibody (HAMA) prior to enrollment (only patients who have received prior therapy with murine antibodies)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Houston Methodist Hospital
Houston, Texas, 77030
Premal Lulla, MD, (713) 441-1450, lulla@bcm.edu
About this study
Official title: Immunotherapy For Adults With GPC3-Positive Solid Tumors Using Interleukin-15 And -21 Armored Glypican-3-Specific Chimeric Antigen Receptor Expressing Autologous T Cells
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.
Read more
In order to get them to kill cancers more effectively, in the laboratory, the study team inserted a new gene called a chimeric antigen receptor (CAR) into T cells that makes them recognize cancer cells and kill them. When inserted, this new CAR T cell can specifically recognize a protein found on solid tumors, called glypican-3 (GPC3). To make this GPC3-CAR more effective, the study team also added two genes called IL15 and IL21 that help CAR T cells grow better and stay in the blood longer so that they may kill tumors better. When the study team did this in the laboratory, they found that this mixture of GPC3-CAR,IL15 and IL21 killed tumor cells better when compared with CAR T cells that did not have IL15 plus IL21 in the laboratory. This study will use those cells, which are called 21.15.GPC3-CAR T cells, to treat patients with solid tumors that have GPC3 on their surface.
The study team also wanted to make sure that they could stop the 21.15.GPC3-CAR T cells from growing in the blood should there be any bad side effects. In order to do so, they inserted a gene called iCasp9 into the CO-EXIST T cells. This allows us the elimination of 21.15.GPC3-CAR T cells in the blood when the gene comes into contact with a medication called AP1903. The drug (AP1903) is an experimental drug that has been tested in humans with no bad side-effects. This drug will only be used to kill the T cells if necessary due to side effects .
The study team has treated patients with T cells that include GPC3. Patients have also been treated with IL-21 and with IL-15. Patients have not been treated with a combination of T cells that contain GPC3, IL-21 and IL-15. To summarize, this study will test the effect of 21.15.GPC3-CAR T cells in patients with solid tumors that express GPC3 on their surface.
The 21.15.GPC3-CAR T cells are an investigational product not yet approved by the Food and Drug Administration.
Read the detailed description
The study team will collect up to 18 teaspoons of patients' blood. This will be used this blood to grow T cells. The study team then grows the T cells and uses a retrovirus (a special virus that can insert the GPC3 CAR and IL15 plus IL21 genes into the T cells) to genetically engineer (generate cells in the laboratory) them. After the CAR and IL15 plus IL21 genes are put into the T cells, the study team ensures that they could kill GPC3 positive solid tumor cells in the laboratory.
LYMPHODEPLETION CHEMOTHERAPY:
Several studies suggest that the infused T cells need room to be able to proliferate and accomplish their functions and that this may not happen if there are too many other T cells in the blood . Lymphodepletion chemotherapy is a way of temporarily decreasing the amount of the body s T cells , so that CO-EXIST T cells can grow better. with the two lymphodepleting chemotherapy drugs we will use are called cyclophosphamide (Cytoxan) and fludarabine. They will both be given daily for 3 days before receiving the T-cell infusion.
WHAT THE INFUSION WILL BE LIKE:
If you agree to participate in this study, at the time you are scheduled to be treated, the cells will be thawed and injected into you over 5 to 10 minutes. You will receive the CO-EXIST T cells 48 to 72 hours after completing the chemotherapy. You may be given Tylenol (acetaminophen) and Benadryl (diphenhydramine) before the CO-EXIST T cells to prevent a possible allergic reaction.
This is a dose escalation study, which means that the study team does now know the highest dose of 21.15.GPC3-CAR T cells that is safe. To find out, the cells will be given to at least 3 participants at a lower dose. If that is safe, the study team will raise the dose level that is given to the next group of participants. The dose a patient received will get will depend on how many participants get the agent before the patient and how they react. The investigator will tell the patient this information. Since the treatment is experimental, what is likely to happen at any dose is not known.
If after 4 weeks the patient's condition does not get worse, the patient may receive another round of lymphodepleting chemotherapy and infusion of 21.15.GPC3-CAR T cells.
All the treatments will be given by the Center for Cell and Gene Therapy at Methodist Hospital of Houston.
Medical tests before treatment:
Before being treated, the patient will receive a series of standard medical tests:
- Physical exam and history
- Blood tests to measure blood cells, kidney and liver function.
- Pregnancy test (if the patient is a female who can get pregnant)
- If the patient is infected with the hepatitis B virus (HBV) sthe study team will do a test to measure the levels of the virus
- Measurements of the patient's tumor by scans (either CT or MRI). Side effects are rare and include risk of developing an unrelated cancer due to radiation exposure from the machine, but this is unlikely.
- the tumor marker alfa-fetoprotein (AFP), if the patient's tumor produces this protein.
Medical tests during and after treatment:
The patient will receive standard medical tests when you are getting the infusions and after:
- Physical exams and History
- Blood tests to measure blood cells, kidney and liver function.
- If the patient is infected with the hepatitis B virus (HBV) the study team will repeat the test and monitor the levels of the virus
- Measurements of the patient's tumor by scans (same scans used before treatment) (4 - 6 weeks after the infusion) and AFP (if applicable at 1, 2, and 4 weeks after the infusion).
- Tumor biopsy of an accessible tumor between 2-4 weeks after the infusion and as clinically indicated thereafter. For additional clinically indicated tumor biopsies the study team will ask for a portion of the sample for research. The study team will not obtain biopsies if the patient's tumors are not accessible.
FOLLOW-UP STUDIES:
The study team will follow the patient during and after the injections. To learn more about the way the T cells are working in the patient's body, up to 60 mL (up to 12 teaspoons) of blood will be taken from the patient before the chemotherapy, before the T-cell infusion, 1 to 4 hours after the infusion, 3 to 4 days after the infusion (this time point is optional ) at 1 week, 2 weeks, 4 weeks and 8 weeks after the injection, every 3 months for 1 year, every 6 months for 4 years and then every year for the next 10 years. Total participation time for this study will be 15 years.
During the time points listed above, if the T cells are found in the patient's blood at a certain amount an extra 5mL of blood may need to be collected for additional testing .
The study team will use this blood to look for the frequency and activity of the cells that we have given; that is, to learn more about the way the T cells are working and how long they last in the body. The study team will also use this blood to see if there are any long-term side effects of putting the new gene (chimeric antigen receptor, CAR) into the cells. In addition to the blood draws, because the patient will receive cells that have had a new gene put in them, the patient will need to have long term follow up for 15 years so the study team can see if there are any long-term side effects of the gene transfer.
Once a year, the patient will be asked to have blood drawn and answer questions about their general health and medical condition. The investigators may ask the patient to report any recent hospitalizations, new medications, or the development of conditions or illness that were not present when the patient enrolled in the study and may request that physical exams and/or laboratory tests be performed if necessary.
When tumor biopsy is performed for clinical reasons the study team will request permission to obtain excess sample to learn more about the effects of the treatment on the patient's disease.
In the event of death, the study team will request permission to perform an autopsy (a procedure where a doctor examines the body to look for a cause of death) to learn more about the effects of the treatment on the patient's disease and if there were any side effects from the cells with the new gene.
In addition, the study team will ask for your permission to use tumor biopsy for research purposes only. Associated risk with the biopsy will be discussed with the patient in detail in a procedure specific consent form. The study team will test the sample to see if the 21.15.GPC3-CAR T cells can be found in the tumor and what effect they had on the tumor cells.
If the patient develops a second abnormal cancer growth, significant blood or nervous system disorder during the trial, the study team will request that a biopsy sample of the tissue will be tested.
The remaining blood and/or tissue samples that are not needed directly for the patient could be used to help researchers learn about this disease and/or immune therapy. These specimens and information about the patient's circumstances may be shared with other cancer researchers. Although there will be a record identifying under what circumstances these specimens were obtained, under all circumstances the patient's identity will be kept confidential. There is a small risk for the loss of confidentiality. However, study personnel will make every effort to minimize this risk. Samples will be kept at Baylor College of Medicine until they are exhausted.
- Conditions
- Hepatoblastoma, Hepatocellular Carcinoma, Wilms Tumor, Malignant Rhabdoid Tumor, Yolk Sac Tumor, Rhabdomyosarcoma
- Being tested
- 21.15.GPC3-CAR T cells
- Keywords
- GPC3-positive tumors, GPC3-CAR T cells
- Medical subject headings
- Hepatoblastoma, Carcinoma, Hepatocellular, Wilms Tumor, Rhabdoid Tumor, Endodermal Sinus Tumor, Rhabdomyosarcoma, Liposarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
21.15.GPC3-CAR T cellsGenetic
Three different dosing schedules will be evaluated.
The following dose levels will be evaluated:
The following dose levels will be evaluated:
DL0: 3x10^7/m2 DL1: 1x10^8/m2 DL2: 3x10^8/m2 DL3: 1x10^9/m2
| Study group | Role | What they receive |
|---|---|---|
| 21.15.GPC3-CAR T cells | Experimental | GPC3-CAR and the IL15 plus IL21 will be administered to patients with GPC3-positive solid tumors. Receives: Genetic: 21.15.GPC3-CAR T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients with Dose Limiting Toxicity
A dose limiting toxicity is defined as any toxicity that is considered to be primarily related to the GPC3-CAR T cells. Specifically those which are Grade 5; non-hematologic Grade 3-4 not returning to Grade 2 within 72 hours; Grade 2-4 allergic reaction; Hematologic Grade 4 that fails to return to Grade 2 or baseline (whichever is more severe) within 14 days; all grade 4 CRS and neurologic toxicities and grade 3 CRS and neurologic toxicities that fail to return to Grade 1 within 7 days
Measured: 4 weeks
Secondary outcomes (2)
Percent of Patients with best response as either complete remission or partial remission
Response rates will be estimated as the percent of patients whose best response is either complete remission or partial remission by combining the data from the two patients. To compare with historical data, a 95% confidence interval will be calculated for the response rate
Measured: 4 weeks
Median T cell persistence
T cell persistence will be measured by PCR
Measured: 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2024
- Study start10 Jun 2025
- Primary completion1 Feb 2028 (estimated)
- Study completion1 Feb 2043 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HepatoblastomaStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, Premal Lulla, Baylor College of Medicine
- Investigators
- Premal Lulla, MD, Principal investigator, Baylor College of MedicineDavid Steffin, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06198296
- Sponsor's protocol ID
- H-54859 CO-EXIST
- Status last verified
- Oct 2025
- First submitted
- 27 Dec 2023
- Last updated
- 3 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immunotherapy For Adults With GPC3-Positive Solid Tumors Using Interleukin-15 And -21 Armored Glypican-3-Specific Chimeric Antigen Receptor Expressing Autologous T Cells
- ClinicalTrials.gov ID
- NCT06198296
- Acronym / sponsor ID
- H-54859 CO-EXIST
- Conditions
- Hepatoblastoma, Hepatocellular Carcinoma, Wilms Tumor, Malignant Rhabdoid Tumor, Yolk Sac Tumor, Rhabdomyosarcoma
- Keywords
- GPC3-positive tumors, GPC3-CAR T cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- 10 Jun 2025 (actual)
- Primary completion
- 1 Feb 2028 (estimated)
- Study completion
- 1 Feb 2043 (estimated)
- First posted
- 10 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
In order to get them to kill cancers more effectively, in the laboratory, the study team inserted a new gene called a chimeric antigen receptor (CAR) into T cells that makes them recognize cancer cells and kill them. When inserted, this new CAR T cell can specifically recognize a protein found on solid tumors, called glypican-3 (GPC3). To make this GPC3-CAR more effective, the study team also added two genes called IL15 and IL21 that help CAR T cells grow better and stay in the blood longer so that they may kill tumors better. When the study team did this in the laboratory, they found that this mixture of GPC3-CAR,IL15 and IL21 killed tumor cells better when compared with CAR T cells that did not have IL15 plus IL21 in the laboratory. This study will use those cells, which are called 21.15.GPC3-CAR T cells, to treat patients with solid tumors that have GPC3 on their surface.
The study team also wanted to make sure that they could stop the 21.15.GPC3-CAR T cells from growing in the blood should there be any bad side effects. In order to do so, they inserted a gene called iCasp9 into the CO-EXIST T cells. This allows us the elimination of 21.15.GPC3-CAR T cells in the blood when the gene comes into contact with a medication called AP1903. The drug (AP1903) is an experimental drug that has been tested in humans with no bad side-effects. This drug will only be used to kill the T cells if necessary due to side effects .
The study team has treated patients with T cells that include GPC3. Patients have also been treated with IL-21 and with IL-15. Patients have not been treated with a combination of T cells that contain GPC3, IL-21 and IL-15. To summarize, this study will test the effect of 21.15.GPC3-CAR T cells in patients with solid tumors that express GPC3 on their surface.
The 21.15.GPC3-CAR T cells are an investigational product not yet approved by the Food and Drug Administration.
Detailed description
The study team will collect up to 18 teaspoons of patients' blood. This will be used this blood to grow T cells. The study team then grows the T cells and uses a retrovirus (a special virus that can insert the GPC3 CAR and IL15 plus IL21 genes into the T cells) to genetically engineer (generate cells in the laboratory) them. After the CAR and IL15 plus IL21 genes are put into the T cells, the study team ensures that they could kill GPC3 positive solid tumor cells in the laboratory.
LYMPHODEPLETION CHEMOTHERAPY:
Several studies suggest that the infused T cells need room to be able to proliferate and accomplish their functions and that this may not happen if there are too many other T cells in the blood . Lymphodepletion chemotherapy is a way of temporarily decreasing the amount of the body s T cells , so that CO-EXIST T cells can grow better. with the two lymphodepleting chemotherapy drugs we will use are called cyclophosphamide (Cytoxan) and fludarabine. They will both be given daily for 3 days before receiving the T-cell infusion.
WHAT THE INFUSION WILL BE LIKE:
If you agree to participate in this study, at the time you are scheduled to be treated, the cells will be thawed and injected into you over 5 to 10 minutes. You will receive the CO-EXIST T cells 48 to 72 hours after completing the chemotherapy. You may be given Tylenol (acetaminophen) and Benadryl (diphenhydramine) before the CO-EXIST T cells to prevent a possible allergic reaction.
This is a dose escalation study, which means that the study team does now know the highest dose of 21.15.GPC3-CAR T cells that is safe. To find out, the cells will be given to at least 3 participants at a lower dose. If that is safe, the study team will raise the dose level that is given to the next group of participants. The dose a patient received will get will depend on how many participants get the agent before the patient and how they react. The investigator will tell the patient this information. Since the treatment is experimental, what is likely to happen at any dose is not known.
If after 4 weeks the patient's condition does not get worse, the patient may receive another round of lymphodepleting chemotherapy and infusion of 21.15.GPC3-CAR T cells.
All the treatments will be given by the Center for Cell and Gene Therapy at Methodist Hospital of Houston.
Medical tests before treatment:
Before being treated, the patient will receive a series of standard medical tests:
- Physical exam and history
- Blood tests to measure blood cells, kidney and liver function.
- Pregnancy test (if the patient is a female who can get pregnant)
- If the patient is infected with the hepatitis B virus (HBV) sthe study team will do a test to measure the levels of the virus
- Measurements of the patient's tumor by scans (either CT or MRI). Side effects are rare and include risk of developing an unrelated cancer due to radiation exposure from the machine, but this is unlikely.
- the tumor marker alfa-fetoprotein (AFP), if the patient's tumor produces this protein.
Medical tests during and after treatment:
The patient will receive standard medical tests when you are getting the infusions and after:
- Physical exams and History
- Blood tests to measure blood cells, kidney and liver function.
- If the patient is infected with the hepatitis B virus (HBV) the study team will repeat the test and monitor the levels of the virus
- Measurements of the patient's tumor by scans (same scans used before treatment) (4 - 6 weeks after the infusion) and AFP (if applicable at 1, 2, and 4 weeks after the infusion).
- Tumor biopsy of an accessible tumor between 2-4 weeks after the infusion and as clinically indicated thereafter. For additional clinically indicated tumor biopsies the study team will ask for a portion of the sample for research. The study team will not obtain biopsies if the patient's tumors are not accessible.
FOLLOW-UP STUDIES:
The study team will follow the patient during and after the injections. To learn more about the way the T cells are working in the patient's body, up to 60 mL (up to 12 teaspoons) of blood will be taken from the patient before the chemotherapy, before the T-cell infusion, 1 to 4 hours after the infusion, 3 to 4 days after the infusion (this time point is optional ) at 1 week, 2 weeks, 4 weeks and 8 weeks after the injection, every 3 months for 1 year, every 6 months for 4 years and then every year for the next 10 years. Total participation time for this study will be 15 years.
During the time points listed above, if the T cells are found in the patient's blood at a certain amount an extra 5mL of blood may need to be collected for additional testing .
The study team will use this blood to look for the frequency and activity of the cells that we have given; that is, to learn more about the way the T cells are working and how long they last in the body. The study team will also use this blood to see if there are any long-term side effects of putting the new gene (chimeric antigen receptor, CAR) into the cells. In addition to the blood draws, because the patient will receive cells that have had a new gene put in them, the patient will need to have long term follow up for 15 years so the study team can see if there are any long-term side effects of the gene transfer.
Once a year, the patient will be asked to have blood drawn and answer questions about their general health and medical condition. The investigators may ask the patient to report any recent hospitalizations, new medications, or the development of conditions or illness that were not present when the patient enrolled in the study and may request that physical exams and/or laboratory tests be performed if necessary.
When tumor biopsy is performed for clinical reasons the study team will request permission to obtain excess sample to learn more about the effects of the treatment on the patient's disease.
In the event of death, the study team will request permission to perform an autopsy (a procedure where a doctor examines the body to look for a cause of death) to learn more about the effects of the treatment on the patient's disease and if there were any side effects from the cells with the new gene.
In addition, the study team will ask for your permission to use tumor biopsy for research purposes only. Associated risk with the biopsy will be discussed with the patient in detail in a procedure specific consent form. The study team will test the sample to see if the 21.15.GPC3-CAR T cells can be found in the tumor and what effect they had on the tumor cells.
If the patient develops a second abnormal cancer growth, significant blood or nervous system disorder during the trial, the study team will request that a biopsy sample of the tissue will be tested.
The remaining blood and/or tissue samples that are not needed directly for the patient could be used to help researchers learn about this disease and/or immune therapy. These specimens and information about the patient's circumstances may be shared with other cancer researchers. Although there will be a record identifying under what circumstances these specimens were obtained, under all circumstances the patient's identity will be kept confidential. There is a small risk for the loss of confidentiality. However, study personnel will make every effort to minimize this risk. Samples will be kept at Baylor College of Medicine until they are exhausted.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 21.15.GPC3-CAR T cells | Experimental | Genetic: 21.15.GPC3-CAR T cellsGPC3-CAR and the IL15 plus IL21 will be administered to patients with GPC3-positive solid tumors. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 21.15.GPC3-CAR T cellsThree different dosing schedules will be evaluated. The following dose levels will be evaluated: The following dose levels will be evaluated: DL0: 3x10^7/m2 DL1: 1x10^8/m2 DL2: 3x10^8/m2 DL3: 1x10^9/m2 | Genetic | 21.15.GPC3-CAR T cells |
Primary outcome
Number of Patients with Dose Limiting Toxicity
Time frame: 4 weeks
A dose limiting toxicity is defined as any toxicity that is considered to be primarily related to the GPC3-CAR T cells. Specifically those which are Grade 5; non-hematologic Grade 3-4 not returning to Grade 2 within 72 hours; Grade 2-4 allergic reaction; Hematologic Grade 4 that fails to return to Grade 2 or baseline (whichever is more severe) within 14 days; all grade 4 CRS and neurologic toxicities and grade 3 CRS and neurologic toxicities that fail to return to Grade 1 within 7 days
2 secondary outcomes
Percent of Patients with best response as either complete remission or partial remission
Time frame: 4 weeks
Median T cell persistence
Time frame: 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Premal Lulla, MD principal investigator
Baylor College of Medicine
David Steffin, MD principal investigator
Baylor College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 21.15.GPC3-CAR T cells in people with Hepatoblastoma and Hepatocellular Carcinoma, run by Baylor College of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Premal Lulla, MD ((713) 441-1450, lulla@bcm.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients with Dose Limiting Toxicity (time frame: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2025.