NeuroblastomaPhase 1NESTLESNCT00085930
Blood T-Cells and EBV Specific CTLs Expressing GD-2 Specific Chimeric T Cell Receptors to Neuroblastoma Patients
A Phase 1 study testing EBV specific CTLs in people with Neuroblastoma, run by Baylor College of Medicine at 1 site in United States.
Trial summary
Patients have high-risk neuroblastoma, a form of cancer typically found in children. The patients previously participated in a gene transfer research study using special immune cells. This research study combines two different ways of fighting disease, antibodies and T cells. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- EBV specific CTLs
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2003to Aug 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
Who may be able to join
- High risk neuroblastoma with a history of persistent or relapsed disease, or after initial therapy
- Patients must have a life expectancy of at least 12 weeks
- Patients must have recovered from the toxic effects of all prior chemotherapy before entering this study
- Patients must not be currently receiving any investigational agents or have not received any tumor vaccines within the previous 6 weeks
- Patients must have an ANC > 500, platelet count > 20,000
- Patients who have received prior therapy with murine antibodies must have documentation of absence of human anti-mouse antibodies (HAMA) prior to enrollment on this study
Show all 19Show fewer
- Patients must have bilirubin less than 3 times the upper limit of normal
- Patients must have AST less than 5 times the upper limit of normal
- Patients must have serum creatinine less than 3 times upper limit of normal
- Patient may not have cardiomegaly or bilateral pulmonary infiltrates on chest radiograph. Patients may have pulmonary metastatic lesions
- Patient may not have an oxygen requirement as defined by pulse oximetry of > 90% on room air
- Patients must have Karnofsky score of > 60% if > 10 years old or Lansky performance score of greater than 60% if 10 years old or younger
- Patients must have autologous transduced EBV-specific CTLs and transduced peripheral blood T-cells with 15% expression or greater of 14g2a.zeta determined by flow-cytometry
- Sexually active patients must be willing to utilize one of the more effective birth control methods during the study and for 3 months after the study is concluded. The male partner should use a condom
- Patients must not be pregnant or lactating
- Patients must not have tumor in a location where enlargement could cause airway obstruction
- Patients must not have a history of hypersensitivity to murine protein-containing products
- Patients must not have a known sensitivity to rat monoclonal antibodies
- Note: All labs must be collected within 10 days prior to initiation of study related treatment
Who cannot join
- Patients not meeting eligibility criteria
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Administration of Peripheral Blood T-Cells and EBV Specific CTLs Transduced to Express GD-2 Specific Chimeric T Cell Receptors to Patients With Neuroblastoma
Patients have high-risk neuroblastoma, a form of cancer typically found in children. The patients previously participated in a gene transfer research study using special immune cells. This research study combines two different ways of fighting disease, antibodies and T cells. Antibodies are types of proteins that protect the body from bacterial and other infections. T cells, also called cytotoxic T lymphocytes or CTLs, are special infection-fighting blood cells that can kill some tumor cells. Both antibodies and T cells have been used to treat patients with cancers and while they have shown promise, they have not been strong enough to cure most patients. The antibody used in this study is called 14g2a. This antibody sticks to neuroblastoma cells because of a substance on the outside of these cells called GD2. 14g2a and other antibodies that bind to GD2 have been used to treat people with neuroblastoma. For this study 14g2a has been changed so that instead of floating free in the blood, it is now joined to T cells. When an antibody is joined to a T cell in this way it is called a chimeric receptor. T lymphocytes or CTLs can kill tumor cells but there normally are not enough of them to kill all tumor cells. Some researchers have taken T cells from a person's blood, grown more of them in the laboratory and then given them back to the patient. Sometimes an antibody or chimeric receptor is attached to these T cells to help them bind to tumor cells. These chimeric receptor-T cells seem to kill some of the tumor, but they don't last very long in the body and so the tumor eventually comes back. We have found that T cells that are also trained to recognize the virus that causes infectious mononucleosis, Epstein Barr Virus (EBV), can stay in the blood stream for many years. By joining the 14g2a antibody to the CTLs that recognize EBV, we believe we will make a cell that can last a long time in the body (because they are EBV-specific) and recognize and kill neuroblastoma cells (because an antibody that can recognize these cells has been placed on their surface). Patients received treatment with the immune cells described above. They may want to receive an additional dose of these cells. This is being offered as an option because their neuroblastoma has returned and they have enough cells remaining to provide the patients with an additional dose. These 14g2a antibody CTLs are an investigational product not approved by the Food and Drug Administration.
Read the detailed description
Earlier, the patient gave us blood to make 14g2a chimeric receptor-T cells and 14g2a chimeric receptor-EBV CTLs in the laboratory. These cells were grown and frozen for the patient. The patient received treatment with these cells. However, there are enough cells remaining that we are able to offer to treat the patient with an additional dose if they would like.
As with the original treatment, the patient will be given an injection of cells into the vein through and IV. Before the injection is received, the patient will be given a dose of Benadryl and Tylenol. The injection will take about 2 minutes. We will follow the patient in the clinic after the injection for 3 hours. The treatment will be given by the Center for Cell and Gene Therapy at Texas Children's Hospital.
Medical tests before treatment--
Before being treated, the patient will receive a series of standard medical tests:
- Physical exam
- Blood tests to measure blood cells, kidney and liver function
- Measurements of tumor by scans and bone marrow studies if the bone marrow may show evidence of disease
Medical tests during and after treatment:
The patient will receive standard medical tests when they are getting the infusions and after:
- Physical exams
- Blood tests to measure blood cells, kidney and liver function
- Measurements of tumor by scans and bone marrow studies if the bone marrow had evidence of disease 6 weeks after each infusion
Because the patient has received cells with a new gene in them the patient will be followed for a total of 15 years to see if there are any long term side effects of gene transfer. If they have a procedure related to their tumor (for example a biopsy or tumor resection), we will request permission to obtain a tissue sample. This will help investigators learn more about T cell and CTL treatment of neuroblastoma. In the event of death, we will request permission to perform an autopsy to learn more about the effects of these infusions on the disease.
- Conditions
- Neuroblastoma
- Being tested
- EBV specific CTLs
- Keywords
- Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EBV specific CTLsBiological
Also called 14g2a.zeta Chimeric Recep Transduc (EBV-CTL) w/o Lymphodepl
CTLs: 2x10e7 cells/m2
| Study group | Role | What they receive |
|---|---|---|
| EBV specific CTLs w/out lymphodepletion | Experimental | Escalating doses of 14g2a.zeta chimeric receptor transduced autologous EBV specific cytotoxic T-lymphocytes (EBV-CTL) and 14g2a.zeta transduced autologous peripheral blood T-cells administered to patients with Neuroblastoma. Receives: Biological: EBV specific CTLs |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the safety of escalating doses of 14g2a.zeta chimeric receptor transduced autologous EBV specific cytotoxic T-lymphocytes (EBV-CTL) and 14g2a.zeta transduced autologous peripheral blood T-cells
Listings of adverse events by patients will include the time to onset, the duration of each event, the severity of each event, and the relationship of the event to study therapy, whether it was a serious event, and whether it caused withdrawal.
Measured: 15 yrs
Secondary outcomes (3)
Determine the differential survival and function of these two infused cell-types in vivo, in particular to determine if chimeric receptor transduced EBV-CTLs survive longer than transduced peripheral-blood T-cells.
To compare the in vivo survival and expansion of the T cells and the CTL and to determine if the T cells or the CTL expand more rapidly after infusion.
Measured: 15 yrs
Determine anti-tumor effects of transduced peripheral blood T-cells and EBV specific CTLs in vivo.
Frequencies and proportions of anti-tumor activity based on evaluations of tumor size.
Measured: 15 years
Compare the differential survival of these infused cells in an additional 6 patients treated at dose level #1 without CD45 antibody mediated lymphodepletion, to patients previously treated at dose levels #2 and #3.
Detect a 1-week half-life difference between dose level 1 and dose level 2
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 posted21 Jun 2004 (estimated)
- Study startApr 2003
- Primary completionJan 2010
- Study completionAug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for NeuroblastomaStudies 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
- 19 participants (actual)
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
- Sponsor
- Investigators
- Helen E Heslop, MD, Study director, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00085930
- Sponsor's protocol ID
- 13149-NESTLES
- Other IDs
- NESTLES
- Acronym
- NESTLES
- Status last verified
- Dec 2025
- First submitted
- 17 Jun 2004
- Last updated
- 7 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Li CH, Sharma S, Heczey AA, Woods ML, Steffin DHM, Louis CU, Grilley BJ, Thakkar SG, Wu M, Wang T, Rooney CM, Brenner MK, Heslop HE. Long-term outcomes of GD2-directed CAR-T cell therapy in patients with neuroblastoma. Nat Med. 2025 Apr;31(4):1125-1129. doi: 10.1038/s41591-025-03513-0. Epub 2025 Feb 17.
PubMed 39962287Louis CU, Savoldo B, Dotti G, Pule M, Yvon E, Myers GD, Rossig C, Russell HV, Diouf O, Liu E, Liu H, Wu MF, Gee AP, Mei Z, Rooney CM, Heslop HE, Brenner MK. Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. Blood. 2011 Dec 1;118(23):6050-6. doi: 10.1182/blood-2011-05-354449. Epub 2011 Oct 7.
PubMed 21984804
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Administration of Peripheral Blood T-Cells and EBV Specific CTLs Transduced to Express GD-2 Specific Chimeric T Cell Receptors to Patients With Neuroblastoma
- ClinicalTrials.gov ID
- NCT00085930
- Acronym / sponsor ID
- NESTLES / 13149-NESTLES
- Conditions
- Neuroblastoma
- Keywords
- Neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Apr 2003
- Primary completion
- Jan 2010 (actual)
- Study completion
- Aug 2025 (actual)
- First posted
- 21 Jun 2004 (estimated)
- 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.
Patients have high-risk neuroblastoma, a form of cancer typically found in children. The patients previously participated in a gene transfer research study using special immune cells. This research study combines two different ways of fighting disease, antibodies and T cells. Antibodies are types of proteins that protect the body from bacterial and other infections. T cells, also called cytotoxic T lymphocytes or CTLs, are special infection-fighting blood cells that can kill some tumor cells. Both antibodies and T cells have been used to treat patients with cancers and while they have shown promise, they have not been strong enough to cure most patients. The antibody used in this study is called 14g2a. This antibody sticks to neuroblastoma cells because of a substance on the outside of these cells called GD2. 14g2a and other antibodies that bind to GD2 have been used to treat people with neuroblastoma. For this study 14g2a has been changed so that instead of floating free in the blood, it is now joined to T cells. When an antibody is joined to a T cell in this way it is called a chimeric receptor. T lymphocytes or CTLs can kill tumor cells but there normally are not enough of them to kill all tumor cells. Some researchers have taken T cells from a person's blood, grown more of them in the laboratory and then given them back to the patient. Sometimes an antibody or chimeric receptor is attached to these T cells to help them bind to tumor cells. These chimeric receptor-T cells seem to kill some of the tumor, but they don't last very long in the body and so the tumor eventually comes back. We have found that T cells that are also trained to recognize the virus that causes infectious mononucleosis, Epstein Barr Virus (EBV), can stay in the blood stream for many years. By joining the 14g2a antibody to the CTLs that recognize EBV, we believe we will make a cell that can last a long time in the body (because they are EBV-specific) and recognize and kill neuroblastoma cells (because an antibody that can recognize these cells has been placed on their surface). Patients received treatment with the immune cells described above. They may want to receive an additional dose of these cells. This is being offered as an option because their neuroblastoma has returned and they have enough cells remaining to provide the patients with an additional dose. These 14g2a antibody CTLs are an investigational product not approved by the Food and Drug Administration.
Detailed description
Earlier, the patient gave us blood to make 14g2a chimeric receptor-T cells and 14g2a chimeric receptor-EBV CTLs in the laboratory. These cells were grown and frozen for the patient. The patient received treatment with these cells. However, there are enough cells remaining that we are able to offer to treat the patient with an additional dose if they would like.
As with the original treatment, the patient will be given an injection of cells into the vein through and IV. Before the injection is received, the patient will be given a dose of Benadryl and Tylenol. The injection will take about 2 minutes. We will follow the patient in the clinic after the injection for 3 hours. The treatment will be given by the Center for Cell and Gene Therapy at Texas Children's Hospital.
Medical tests before treatment--
Before being treated, the patient will receive a series of standard medical tests:
- Physical exam
- Blood tests to measure blood cells, kidney and liver function
- Measurements of tumor by scans and bone marrow studies if the bone marrow may show evidence of disease
Medical tests during and after treatment:
The patient will receive standard medical tests when they are getting the infusions and after:
- Physical exams
- Blood tests to measure blood cells, kidney and liver function
- Measurements of tumor by scans and bone marrow studies if the bone marrow had evidence of disease 6 weeks after each infusion
Because the patient has received cells with a new gene in them the patient will be followed for a total of 15 years to see if there are any long term side effects of gene transfer. If they have a procedure related to their tumor (for example a biopsy or tumor resection), we will request permission to obtain a tissue sample. This will help investigators learn more about T cell and CTL treatment of neuroblastoma. In the event of death, we will request permission to perform an autopsy to learn more about the effects of these infusions on the disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| EBV specific CTLs w/out lymphodepletion | Experimental | Biological: EBV specific CTLsEscalating doses of 14g2a.zeta chimeric receptor transduced autologous EBV specific cytotoxic T-lymphocytes (EBV-CTL) and 14g2a.zeta transduced autologous peripheral blood T-cells administered to patients with Neuroblastoma. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- High risk neuroblastoma with a history of persistent or relapsed disease, or after initial therapy
- Patients must have a life expectancy of at least 12 weeks
- Patients must have recovered from the toxic effects of all prior chemotherapy before entering this study
- Patients must not be currently receiving any investigational agents or have not received any tumor vaccines within the previous 6 weeks
- Patients must have an ANC > 500, platelet count > 20,000
Key exclusion criteria (1 listed)
- Patients not meeting eligibility criteria
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 |
|---|---|---|
| EBV specific CTLsAlso: 14g2a.zeta Chimeric Recep Transduc (EBV-CTL) w/o LymphodeplCTLs: 2x10e7 cells/m2 | Biological | EBV specific CTLs w/out lymphodepletion |
Primary outcome
Evaluate the safety of escalating doses of 14g2a.zeta chimeric receptor transduced autologous EBV specific cytotoxic T-lymphocytes (EBV-CTL) and 14g2a.zeta transduced autologous peripheral blood T-cells
Time frame: 15 yrs
Listings of adverse events by patients will include the time to onset, the duration of each event, the severity of each event, and the relationship of the event to study therapy, whether it was a serious event, and whether it caused withdrawal.
3 secondary outcomes
Determine the differential survival and function of these two infused cell-types in vivo, in particular to determine if chimeric receptor transduced EBV-CTLs survive longer than transduced peripheral-blood T-cells.
Time frame: 15 yrs
Determine anti-tumor effects of transduced peripheral blood T-cells and EBV specific CTLs in vivo.
Time frame: 15 years
Compare the differential survival of these infused cells in an additional 6 patients treated at dose level #1 without CD45 antibody mediated lymphodepletion, to patients previously treated at dose levels #2 and #3.
Time frame: 15 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2026.
- Derived from this study: PubMed 39962287Li CH, Sharma S, Heczey AA, Woods ML, Steffin DHM, Louis CU, Grilley BJ, Thakkar SG, Wu M, Wang T, Rooney CM, Brenner MK, Heslop HE. Long-term outcomes of GD2-directed CAR-T cell therapy in patients with neuroblastoma. Nat Med. 2025 Apr;31(4):1125-1129. doi: 10.1038/s41591-025-03513-0. Epub 2025 Feb 17.
- Derived from this study: PubMed 21984804Louis CU, Savoldo B, Dotti G, Pule M, Yvon E, Myers GD, Rossig C, Russell HV, Diouf O, Liu E, Liu H, Wu MF, Gee AP, Mei Z, Rooney CM, Heslop HE, Brenner MK. Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. Blood. 2011 Dec 1;118(23):6050-6. doi: 10.1182/blood-2011-05-354449. Epub 2011 Oct 7.
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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. This study is not recruiting at the moment.
Helen E Heslop, MD study director
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 EBV specific CTLs in people with Neuroblastoma, 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?
The sponsor lists 19 inclusion and 1 exclusion criteria. 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: finished, as of the sponsor's last update on 7 Jan 2026. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the safety of escalating doses of 14g2a.zeta chimeric receptor transduced autologous EBV specific cytotoxic T-lymphocytes (EBV-CTL) and 14g2a.zeta transduced autologous peripheral blood T-cells (time frame: 15 yrs). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00085930, which also lists 2 linked publications.
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 7 Jan 2026.