ClinicalTrials.gov record NCT03294954

GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

GD2 Specific Chimeric Antigen Receptor (CAR) and Interleukin-15 Expressing Autologous Natural Killer T-cells to Treat Children With Neuroblastoma

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age1 year old to 21 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Phase 1 dose-finding study of autologous NKT cells engineered with a GD2-specific CAR and an Interleukin-15 gene (called GINAKIT cells) for children and young people with relapsed or refractory neuroblastoma. Participants provide blood to make the cells, receive short-course lymphodepleting chemotherapy, then an IV infusion of GINAKIT cells; some dose levels add weekly Etanercept. The study seeks the highest safe dose, measures how long the cells persist in the blood and their effect on tumors, and includes long-term safety follow-up.

What it is studying. It is testing the safety and dose limits of autologous GD2-CAR, Interleukin-15–expressing NKT cell infusions (GINAKIT), how long those cells can be detected in blood, and tumor responses with and without Etanercept.

What the researchers hope to learn. Primary scientific goal: identify the maximum tolerated dose and dose-limiting toxicities of autologous GD2-specific CAR and IL-15–expressing NKT cells given with Etanercept. Secondary goals: describe anti-tumor responses, compare responses with and without Etanercept, measure persistence of infused cells in blood, and monitor long-term safety after gene transfer for up to 15 years.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This research study combines two different ways of fighting cancer: antibodies and Natural Killer T cells (NKT). Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special white 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. 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 a previous clinical trial, investigators made artificial genes called a chimeric antigen receptors (CAR), from an antibody called 14g2a that recognizes GD2, a molecule found on almost all neuroblastoma cells (GD2-CAR). Investigators put these genes into the patients' own T cells and gave them back to patients that had neuroblastoma. NKT cells are another special subgroup of white blood cells that can specifically go into tumor tissue of neuroblastoma. Inside the tumor, there are other white blood cells called macrophages which help the cancer cells to grow and recover from injury. NKT cells can specifically kill these macrophages and slow the tumor growth. We will expand NKT cells and add GD2-specific chimeric antigen receptors to the cells. We think these cells might be better able to attack NB since they also work by destroying the macrophages that allows the tumor to grow. The chimeric antigen receptor will also contain a gene segment to make the NKT cells last longer. This gene segment is called CD28. In addition, to further improve the antitumor activity of the GINAKIT cells we added another gene expressing a molecule called Interleukin -15 (IL-15). The combination of these 3 components showed the most antitumor activity by CAR expressing NKT cells and improved these cells' survival in animal models. We also found that a medicine called ETANercept can slow down neuroblastoma growth, which might enhance the effects of the modified cells. In this part of our study, we aim to treat children with hard-to-treat neuroblastoma using these modified NKT cells along with ETANercept. Though ETANercept has been used to treat other diseases, such as rheumatoid arthritis in children, there is limited information about the safety, efficacy, and risk of ETANercept treatment in combination with cellular therapies. GD2-CAR expressing NKTs have not been tested in patients so far. The purpose of this study is to find the largest effective and safe dose of GD2-CAR NKT cells (GINAKIT cells), to evaluate their effect on the tumor and how long they can be detected in the patient's blood and what affect they have on the patient's neuroblastoma.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological
  • GINAKIT cells + Etanercept
Genetic
  • GINAKIT Cells

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Procurement Inclusion Criteria:
  • Relapsed or refractory high risk neuroblastoma
  • Life expectancy of at least 12 weeks
  • Age greater than 1 year and less than 21 years old
  • Karnofsky/Lansky score of 60% or greater
  • Absence of HAMA prior to enrollment (only in patients that have been previously treated with murine antibodies)
  • Ability to tolerate leukocyte apheresis
  • Informed consent and assent (as applicable) obtained from parent/guardian and child.
  • Patients must have an ANC greater than or equal to 500/µl, platelet count greater than or equal to 20,000/µl. Patients may be transfused to obtain a platelet count greater than or equal to 20,000/µl.
  • Pulse Ox greater than or equal to 90% on room air
  • Serum AST less than 3 times the upper limit of normal
  • Total Bilirubin less than 1.5 times the upper limit of normal
  • Creatinine < 1.5 times the upper limit of normal
  • Recovered from the acute toxic effects of all prior chemotherapy based on the enrolling physician's assessment (if some effects of chemotherapy are expected to last long term, patient is eligible if meeting other eligibility criteria).
  • Weight greater than 12kg
  • Negative QuantiFERON-TB or T-SPOT testing within 3 months prior to procurement
  • Procurement

Exclusion criteria

  • Rapidly progressive disease
  • History or hypersensitivity to murine protein-containing products
  • Tumor causing airway obstruction
  • Currently receiving immunosuppressive drugs such as corticosteroids, tacrolimus or cyclosporine
  • Severe previous toxicity from cyclophosphamide or fludarabine based on the enrolling physician's assessment
  • HIV infection
  • History of hypersensitivity, anaphylaxis, and/or adverse event with Etanercept
  • Treatment Inclusion Criteria:
  • Relapsed or refractory high risk neuroblastoma
  • Life expectancy of at least 12 weeks
  • Age greater than 1 year and less than 21 years old
  • Karnofsky/Lansky score of 60% or greater
  • Patients must have an ANC greater than or equal to 500/µl, platelet count greater than or equal to 20,000/µl. Patients may be transfused to obtain a platelet count greater than or equal to 20,000/µl.
  • Pulse Ox greater than or equal to 90% on room air
  • Serum AST less than 3 times the upper limit of normal
  • Total Bilirubin less than 1.5 times the upper limit of normal
  • Creatinine < 1.5 times the upper limit of normal
  • Recovered from the acute toxic effects of all prior chemotherapy based on the enrolling physician's assessment (if some effects of chemotherapy are expected to last long term, patient is eligible if meeting other eligibility criteria and expected to tolerate lymphodepletion).
  • Absence of human anti-mouse antibodies (HAMA) prior to enrollment for patients who have received prior therapy with murine antibodies
  • Patients must have autologous transduced NKTs with greater than or equal to 20% expression of GD2-specific CAR.
  • Informed consent and assent (as applicable) obtained from parent/guardian and child.
  • Weight greater than 12kg
  • Negative QuantiFERON-TB or T-SPOT testing within 3 months prior to the start of LD
  • Treatment Exclusion Criteria:
  • Rapidly progressive disease
  • Currently receiving any investigational drugs
  • History or hypersensitivity to murine protein-containing products
  • Cardiomegaly or bilateral pulmonary infiltrates on chest radiograph or CT. However, patients with cardiomegaly on imaging may be enrolled if they have an assessment of cardiac function (i.e., ECHO or MUGA) within 3 weeks of starting protocol therapy that is within normal limits. Additionally, patients with bilateral pulmonary infiltrates on imaging may be enrolled if the lesions are not consistent with active neuroblastoma (i.e., negative on functional imaging with PET or MIBG, or by pathologic assessment).
  • Tumor potentially causing airway obstruction
  • Pregnancy or lactation or not willing to use birth control
  • Currently receiving immunosuppressive drugs such as corticosteroids, tacrolimus or cyclosporine
  • Severe previous toxicity form cyclophosphamide or fludarabine based on the enrolling physician's assessment
  • HIV infection
  • History of hypersensitivity, anaphylaxis, and/or adverse event with Etanercept

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

In this study the first step is to collect blood from the patient to make the GINAKIT cells. Once the GINAKIT cells are made they will be administered to the patient. Patients will be treated on a single fixed dose level with Etanercept from now on. If there is severe toxicity related to the combination of GINAKIT cells and Etanercept, we will treat patients on lower dose level. Thus maximum 36 patients will be recruited on the on the combination therapy, including 18 patients potentially at the lower dose level. If we are not able to generate sufficient GINAKIT cells on the fixed dose level, we will treat you with the highest dose level that is achieved. Before getting the GINAKIT cells, the patient will receive cyclophosphamide and fludarabine intravenously (through a needle inserted into a vein or a port-a-cath) for 2 days and then fludarabine alone for one more day. The patient will then receive ETANercept subcutaneously (through a needle just under the skin) once per week starting the day after chemotherapy for 5 doses. The patient will receive the GINAKIT cells the day after first dose of ETANercept. The patient will be given an injection of GINAKIT cells into the vein through an IV line at the assigned dose. Before the patient receives the injection, they may be given a dose of Benadryl (diphenhydramine) and Tylenol (acetaminophen). The injection will take up to 10 minutes. The patient will be monitored in the clinic after the injection for about 4 hours. The treatment will be given by the Center for Cell and Gene Therapy at Texas Children's Hospital. The patient will need to stay in Houston for 4 weeks after the infusion so investigators can monitor the patient for side effects. The patient will have follow-up visits (at weeks 1, 2, 3, 4 and 8; months 3, 6, 9, and 12; twice a year for 4 years and then once a year for the next 10 years - for a total of 15 years) and scheduled disease evaluations after the GINAKIT cell infusion (at week 6 and then as clinically needed). If after 4 weeks the patient's condition does not get worse, the patient may receive another round of lymphodepleting chemotherapy and infusion of GINAKIT2 cells. Reinfusion of cells may occur up to three times and the patient will have to meet treatment eligibility criteria at each lymphodepletion start. The patient will also receive ETANercept with the schedule at each time of treatment. Treatment eligibility are characteristics that must be met such as current health status and other items that ensure patients who could be made worse by participating in the study are not exposed to that risk. The eligibility also ensures researchers achieve accurate results Before being treated, the patient will receive a series of standard medical tests as follows:

  • Physical exam
  • Blood tests to measure blood cells, kidney and liver function
  • Blood tests to ensure the patient do not have tuberculosis.
  • Buccal swabs (cheek scraping)
  • Measurements of the patient's tumor by routine imaging studies and bone marrow evaluation within 4 weeks (preferably 2 weeks) before start of lymphodepletion (no other cancer treatment should be given after these studies and the GINAKIT cell infusion). Imaging studies that have been used in the past to best assess the patient's tumor will be used (Computer Tomogram (CT) or Magnetic Resonance Imaging (MRI), and Positron Emission Tomography (PET/CT), and/or MIBG scan). A PET scan is an imaging test that uses radioactive material to look for disease in the body. A MIBG scan is an imaging test that uses radioactive material and a special scanner to find or confirm the presence of neuroblastoma.
  • A urine pregnancy test. If that patient is pregnant both the patient and their parents will be informed of the pregnancy.

The patient will receive standard medical tests when they are getting the infusions and afterwards as follows:

  • Physical exams
  • Blood tests to measure blood cells counts, kidney and liver function.
  • Subcutaneous injection of ETANercept.
  • Measurements of the patient's tumor by routine imaging studies 4-6 weeks after the infusion.
  • Tumor biopsy at 2 weeks and between 4-6 weeks after the infusion and as clinically indicated thereafter. The evaluation at week 2 after the infusion is for research only and is done to see whether or not there are GINAKIT cells in the tumor. For all clinically indicated tumor biopsies a portion of the sample for research will be requested.
  • If a tumor was in the bone marrow at the time of treatment, the procedure will be repeated at 2 weeks and between 4-6 weeks after the infusion and as clinically indicated thereafter. The evaluation at week 2 after the infusion is for research only and is done to see whether or not there are GINAKIT cells in the bone marrow. For all clinically indicated bone marrow collections a portion of the sample for research will be requested.

To learn more about the way the GINAKIT cells are working and how long they last in the body, an extra amount of blood will be obtained before the chemotherapy, on the day of the GINAKIT cell infusion (before and at the end of the infusion), 1, 2, 3, 4 and 8 weeks after the GINAKIT cell infusion, every 3 months for the 1st year, every 6 months for the next 4 years and then once a year for the next 10 years (for a total of 15 years). The amount of blood taken will be based on the patient's weight with up to a maximum of 60 ml (12 teaspoons) of blood to be obtained at any one time. For children, the total amount of blood drawn will not be more than 3 ml (less than 1 teaspoon) per 1 kg of body weight on any given day. This volume is considered safe, but may be decreased if the patient is anemic (have a low red blood cell count). This blood will also be used to monitor the patient's white blood cell levels. In the event the patient's white blood cell counts are high, they will be admitted to the hospital where they will be treated with hydration, steroids or chemotherapy. The study doctor will discuss with the patient the best treatment options. During the time points listed above, if the GINAKIT 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. If the patient has a procedure where tumor samples are obtained, investigators will request a sample to be used for research purposes. If the patient develops a second abnormal growth, significant blood or nervous system disorder during the trial, a biopsy sample of the tissue will be tested (if a sample can be obtained). The patient will receive supportive care for any acute or chronic toxicities, including blood components or antibiotics, and other intervention as appropriate. Because the patient will receive cells with a new gene in them they will be followed for a total of 15 years to see if there are any long term side effects of gene transfer.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

You will donate blood (leukocyte apheresis) so the team can make modified NKT (GINAKIT) cells, receive short lymphodepleting chemotherapy (cyclophosphamide and fludarabine), then get weekly subcutaneous Etanercept for five doses and an IV infusion of the GINAKIT cells with about four hours of clinic monitoring. You must remain near the treating center for about 4 weeks after the infusion and return for scheduled visits and tests (frequent blood draws, tumor imaging, bone marrow checks) including research tumor biopsies at 2 weeks and 4–6 weeks if possible. If eligible, you may have up to three repeat lymphodepletion plus cell infusions; blood samples will be taken often early and then periodically for a total follow‑up of 15 years to monitor the gene‑modified cells. Supportive care or hospital treatment (for example hydration, steroids, antibiotics, or transfusions) will be provided if needed.

How the study is structured. Phase 1, non-randomized, single-group dose-escalation treatment study. Patients undergo blood collection to manufacture autologous NKT cells, receive lymphodepleting chemotherapy, then a short IV infusion of GINAKIT cells at assigned dose. Some dose levels combine the cell infusion with weekly subcutaneous Etanercept for five doses; reinfusion of cells may be possible up to three times if eligibility is met. Participants remain near the treatment center for about 4 weeks after infusion and have scheduled blood tests, imaging, possible tumor biopsies, and long-term safety follow-up for up to 15 years.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Neuroblastoma. Does it apply to my diagnosis?
  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

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Important dates

Study start
Jan 18, 2018
Primary completion
December 2027 (estimated)
Study completion
Aug 10, 2040 (estimated)
Target enrollment
70 participants (estimated)
Last updated on registry
Sep 19, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Baylor College of Medicine
Principal investigator
Gengwen Tian, MD, PhD, Assistant Professor
Baylor College of Medicine
Collaborators
  • Center for Cell and Gene Therapy, Baylor College of Medicine

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

chimeric antigen receptor
A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Relapsed or refractory
A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
Life expectancy
Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
Karnofsky
A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
Informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
ANC
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
eligibility criteria
The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
immunosuppressive
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
corticosteroids
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
adverse event
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
prior therapy
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
CT
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
ECHO
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
PET
A scan in which a small amount of a radioactive tracer is injected so that activity in tissues can be seen. Often combined with a CT or MRI scan.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
Magnetic Resonance Imaging
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
biopsy
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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