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Recurrent Childhood NeuroblastomaPhase 1NCT04539366

Testing a New Immune Cell Therapy, GD2-Targeted Modified T-cells (GD2CART), in Children, Adolescents, and Young Adults With Relapsed/Refractory Osteosarcoma and Neuroblastoma, GD2-CAR PERSIST Trial

A Phase 1 study testing Biopsy Procedure, Biospecimen Collection and Bone Marrow Aspiration in people with Recurrent Childhood Neuroblastoma and Recurrent Childhood Osteosarcoma, run by National Cancer Institute (NCI) at 6 sites in United States.

Suspended

Paused. Enrolment is on hold for now and may resume.

Official record
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Trial summary

This phase I trial investigates the side effects and determines the best dose of an immune cell therapy called GD2CART, as well as how well it works in treating patients with osteosarcoma or neuroblastoma that has come back (relapsed) or does not respond to treatment (refractory). Read more about this study

Who is it for?
Ages Up to 40 years
What is being tested?
Biopsy Procedure, Biospecimen Collection and Bone Marrow Aspiration
Could I receive a placebo?
No, everyone receives an active treatment
How many people
73 participants
Where
6 sitesUnited States
Study dates
25 Jan 2022to 31 Dec 2040
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 40 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Must have histologically confirmed neuroblastoma or osteosarcoma that is recurrent or refractory and for which standard curative measures do not exist or are no longer effective. Must have histologic verification of their disease at diagnosis or at relapse
  • Patients with osteosarcoma in the dose escalation cohort, must have evaluable or measurable disease at enrollment
  • Patients with osteosarcoma in the expansion cohort must have measurable disease by Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) at enrollment
  • Patients with neuroblastoma in the dose escalation or dose the expansion cohort must have:
  • Prior progressive disease OR refractory disease present since diagnosis AND at least one of the following:
  • Evidence of tumor in the bone marrow (BM) less than or equal to 5%
Show all 45Show fewer
  • At least one metaiodobenzylguanidine (MIBG)-avid soft tissue or skeletal site
  • For MIBG-nonavid disease, at least one FDG-PET- positive soft tissue or skeletal site plus past histologic confirmation
  • Progressive disease is defined as any disease progression occurring at any time after the diagnosis of high-risk neuroblastoma. Refractory disease is defined as an incomplete response of high-risk neuroblastoma to all treatments but without disease progression
  • NOTE: Disease assessment will be required within 2 weeks prior to lymphodepleting chemotherapy. Patients will need to meet criteria for having low burden disease prior to the start of lymphodepleting chemotherapy
  • Must be < 40 years of age
  • There is no limit to the number of prior treatment regimens. The following washout periods prior to leukapheresis apply to patients undergoing leukapheresis on this study. If a patient has cryopreserved peripheral blood mononuclear cells (PBMCs) stored, the following washout periods are strongly recommended but not required and the product is useable if it meets the criteria established in this Investigational New Drug (IND)
  • Myelosuppressive chemotherapy: Patients must not have received myelosuppressive chemotherapy within 3 weeks of leukapheresis (6 weeks if prior nitrosourea)
  • Hematopoietic growth factors: At least 7 days must have elapsed since the completion of therapy with a short-acting growth factor. At least 14 days must have elapsed after receiving pegfilgrastim
  • Biological agent, tyrosine kinase inhibitor, targeted agent, metronomic chemotherapy: At least 7 days must have elapsed since the completion of therapy with a biologic agent, tyrosine kinase inhibitor, targeted agent, or metronomic non-myelosuppressive regimen
  • 131I-MIBG or other radioisotope therapy: At least 6 weeks must have elapsed since prior therapy with 131I-MIBG. At least 6 weeks or 10 half-lives (whichever is shorter) must have elapsed since prior therapy with any other radioisotope
  • Monoclonal antibodies and checkpoint inhibitors: At least 3 weeks or 5 half-lives (whichever is shorter) must have elapsed since prior therapy that included a monoclonal antibody or checkpoint inhibitor
  • Radiotherapy (XRT): 3 weeks must have elapsed since XRT; at least 6 weeks if XRT involved central nervous system (CNS) or lung fields; and at least 12 weeks from total-body irradiation (TBI), craniospinal XRT, or XRT involving ≥ 50% bony pelvis, with the exception that there is no time restriction for palliative radiation with minimal bone marrow involvement and the patient has measurable/evaluable disease outside the radiation port or the site of radiation has documented progression
  • Vaccine therapy, anti-GD2 mAb therapy, or therapy with any genetically engineered T cells: Patients may have received previous vaccine therapy, anti-GD2 monoclonal antibody (mAb) therapy, or therapy with any genetically engineered T cells except prior GD2 CAR T cell therapy. At least 3 weeks or 5 half-lives, whichever is shorter, must have elapsed since any prior vaccine or monoclonal antibody therapy. At least 42 days must have elapsed since prior modified T cell, natural killer (NK) cell, or dendritic cell therapy
  • Allogeneic stem cell transplant/infusion: At least 12 weeks must have elapsed since allogeneic stem cell transplant and without evidence of active graft versus host disease (GVHD)
  • Autologous stem cell transplant/infusion: Patients who received an autologous stem cell infusion following myeloablative therapy should be at least 6 weeks from their infusion. Patients who received an autologous stem cell infusion following non-myeloablative therapy do not have a wash-out period; they are eligible once they meet all other eligibility requirements, including recovery from acute side effects
  • Must meet parameters for apheresis per institutional guidelines. (This criterion does not apply to patients with apheresis product or usable T cell product available for use). Cryopreserved PBMCs stored from participation in other institutional cell therapy or cell collection studies or standard of care may be used to generate the cellular product on this study if they meet the criteria established in this IND
  • Patients > 16 years of age must have Karnofsky >= 70%. Patients =< 16 years of age must have Lansky scale ≥ 70%; corresponding to Eastern Cooperative Oncology Group (ECOG) performance status =< 1
  • Leukocytes >= 750/mcL (Patients must not be refractory to transfusions)
  • Absolute neutrophil count (ANC) >= 500/mcL (Patients must not be refractory to transfusions.)
  • Platelets for both patients with and without bone marrow involvement >= 75,000/mcL (transfusion independent defined as no transfusion in prior 7 days) (Patients must not be refractory to transfusions.)
  • Aspartate aminotransferase (AST)(serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT)(serum glutamate pyruvate transaminase [SGPT]) =< 3 x upper limit of normal (ULN) (For the purpose of this study, the ULN for SGOT is 50 U/L and the ULN for SGPT is 45 U/L)
  • Albumin >= 3 g/dL
  • Total bilirubin =< 1.5 x institutional ULN for age. Patients with Gilbert's syndrome are excluded from the requirement of a normal bilirubin and patients will not be excluded if bilirubin elevation is due to tumor involvement. (Patients with Gilbert's syndrome are excluded from this requirement as long as they have a normal direct/conjugated bilirubin). [Gilbert's syndrome is found in 3-10% of the general population, and is characterized by mild, chronic unconjugated hyperbilirubinemia in the absence of liver disease or overt hemolysis]. Note: Adult values will be used for calculating hepatic toxicity and determining eligibility.
  • International normalized ratio (INR) =< 1.5
  • Age, maximum serum creatinine (mg/dL):
  • 1 month to < 6 months: 0.4 (male), 0.4 (female)
  • 6 months to < 1 year: 0.5 (male), 0.5 (female)
  • 1 to < 2 years: 0.6 (male), 0.6 (female)
  • 2 to < 6 years: 0.8 (male), 0.8 (female)
  • 6 to < 10 years: 1 (male), 1 (female)
  • 10 to < 13 years: 1.2 (male), 1.2 (female)
  • 13 to < 16 years: 1.5 (male), 1.2 (female)
  • >= 16 years: 1.7 (male), 1.4 (female) OR Creatinine clearance or glomerular filtration rate (GFR) >= 60 mL/min/1.73 m^2 for patients with levels above institutional normal
  • Cardiac ejection fraction >= 50% or shortening fraction >= 28%. No clinically significant cardiac dysfunction or pericardial effusion as determined by an ECHO. No clinically significant electrocardiogram (ECG) findings
  • Pulmonary status: No clinically significant pleural effusion (i.e., pleural effusion > grade 1). Baseline oxygen saturation > 92% on room air at rest
  • No acute neurotoxicity greater than grade 2 or any acute neurological toxicity that would impact the investigator's ability to evaluate CAR induced neurotoxicity, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible
  • Females of child-bearing potential and males of reproductive potential who are sexually active must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) from the start of study enrollment until 6 months after GD2 CAR T cell infusion (or until the duration of study participation, in the case of patients who start lymphodepleting chemotherapy but do not receive the GD2 CART infusion). Should a female become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
  • Note: Females of childbearing potential are defined as those who are past the onset of menarche and are not surgically sterile (i.e., bilateral salpingectomy, bilateral oophorectomy, complete hysterectomy) or post menopausal
  • All patients >= 18 years of age must be able to give informed consent or if unable to give consent have a legal authorized representative (LAR) who can give consent for the patient. For patients < 18 years old their LAR (i.e., parent or legal guardian) must give informed consent. Pediatric patients will be included in age appropriate discussion and verbal assent will be obtained for those > 7 years of age, when appropriate, according to local policy

Who cannot join

  • Receiving any other current investigational agents
  • History of anaphylactic reactions attributed to anti-GD2 antibodies or to compounds of similar chemical or biologic composition to GD2CART, cyclophosphamide, fludarabine, or other agents used in this study. History of hypersensitivity to dornase alfa, Chinese hamster ovary cell products, or any of the components of pulmozyme
  • Patients who require systemic corticosteroid or other immunosuppressive therapy. (A one-week washout from systemic corticosteroid or other immunosuppressive therapy is permitted.) Use of physiologic doses of corticosteroids (up to 3 mg/m^2/day prednisone equivalent) are permitted. Use of topical, ocular, intra-articular, intra-nasal, or inhaled corticosteroids are permitted
  • Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • History of additional malignancy other than non-melanoma skin cancer or carcinoma in situ (e.g., cervix, bladder, breast) unless untreated and stable or disease free for at least 3 years
  • Patients with central nervous system (CNS) metastases are not eligible unless CNS metastases have been previously treated and sequential imaging shows no evidence for active disease in the CNS.
Show all 12Show fewer
  • CNS disorder such as cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or autoimmune disease with CNS involvement that in the judgement of the investigator may impair the ability to evaluate neurotoxicity
  • Presence of fungal, bacterial, viral, or other infection that is uncontrolled
  • Ongoing infection with human immunodeficiency virus (HIV), hepatitis B (hepatitis B surface antigen [HBsAg] positive), or hepatitis C virus (anti-HCV positive) as the immunosuppression contained in this study will pose unacceptable risk. A history of HIV, hepatitis B, or hepatitis C is permitted if the viral load is undetectable per quantitative polymerase chain reaction (PCR) and/or nucleic acid testing
  • Primary immunodeficiency or history of systemic autoimmune disease (e.g., Crohns, rheumatoid arthritis, systemic lupus) requiring systemic immunosuppression/systemic disease modifying agents within the last 2 years
  • Females of childbearing potential must have a negative serum or urine pregnancy test. Pregnant females are excluded from this study because the effects of autologous GD2CART on the developing human fetus are unknown and because the chemotherapy agents used in this trial (cyclophosphamide and fludarabine) are category D agents with the potential for teratogenic or abortifacient effects. Additionally, because there is an unknown but potential risk for adverse events (AEs) in nursing infants secondary to treatment of the mother with cyclophosphamide/fludarabine, breastfeeding should be discontinued if the mother is treated with cyclophosphamide/fludarabine. These potential risks may also apply to other agents used in this study
  • In the investigator's judgment, unlikely to complete protocol-required study visits or procedures, including follow-up visits, or comply with the study requirements for participation. Or in the investigator's judgment, if the patient is likely to develop significant toxicity and morbidity from CAR-T cell expansion mediated inflammation based on location of tumor site

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

  • University of Wisconsin Carbone Cancer Center - University Hospital

    Madison, Wisconsin, 53792

  • Lucile Packard Children's Hospital Stanford University

    Palo Alto, California, 94304

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: GD2-CAR PERSIST: Production and Engineering of GD2-Targeted, Receptor Modified T Cells (GD2CART) for Osteosarcoma or Neuroblastoma to Increase Systemic Tumor Exposure

This phase I trial investigates the side effects and determines the best dose of an immune cell therapy called GD2CART, as well as how well it works in treating patients with osteosarcoma or neuroblastoma that has come back (relapsed) or does not respond to treatment (refractory). T cells are infection fighting blood cells that can kill tumor cells. The T cells given in this trial will come from the patient and will have a new gene put in them that makes them able to recognize GD2, a protein on the surface of tumor cells. These GD2-specific T cells may help the body's immune system identify and kill GD2 positive tumor cells.

Read the detailed description

PRIMARY OBJECTIVES:

  1. Determine the feasibility of producing T cells modified to express a GD2-specific chimeric antigen receptor (GD2-CAR-expressing autologous T-lymphocytes [GD2CART]) meeting established release criteria using a dasatinib containing culture platform and retroviral vector in the Miltenyi CliniMACS Prodigy (Registered Trademark) system.

II. Determine the safety and maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) via administration of escalating doses of autologous GD2CART in children and young adults with relapsed/refractory osteosarcoma and neuroblastoma following cyclophosphamide-fludarabine based lymphodepletion.

III. Determine clinical activity in a preliminary fashion of autologous GD2CART in children and young adults with relapsed, refractory osteosarcoma and neuroblastoma.

SECONDARY OBJECTIVES:

  1. Measure persistence of adoptively transferred GD2CART and correlate this with antitumor effects.

II. If unacceptable toxicity occurs that is possibly, probably, or definitely related to GD2CART, assess the capacity for rimiducid (AP1903), a dimerizing agent, to mediate clearance of the genetically engineered cells and resolve toxicity.

III. Describe the feasibility and tolerability of a second infusion of GD2CART in select patients.

EXPLORATORY OBJECTIVES:

  1. Compare persistence of GD2CART administered in this trial to that observed in a previous trial using GD2.OX40.28.z.iCasp9 CAR T cells (NCI 14-C-0059) and assess features of the T cell product and the expanded T cells in vivo that correlate with persistence.

II. Conduct exploratory studies measuring levels of circulating myeloid cells including myeloid derived suppressor cells (MDSCs) in patients treated on this trial and compare levels to those observed in NCI 14-C-0059.

III. Explore GD2 expression in patients with neuroblastoma and osteosarcoma, including patients who have previously received anti-GD2 antibodies, from tissue and/or bone marrow samples at study entry and if available, after cell infusion.

OUTLINE: This is a dose-escalation study of GD2CART followed by a dose-expansion study.

LYMPHODEPLETION CHEMOTHERAPY: Patients receive fludarabine phosphate intravenously (IV) daily on days -4 to -2 and cyclophosphamide IV daily on days -3 to -2.

GD2CART: Patients receive GD2CART cells IV on day 0.

Patients also undergo echocardiogram (ECHO), multigated acquisition scan (MUGA) or cardiac magnetic resonance imaging (MRI) scan, blood sample collection throughout the trial, and tumor biopsies and bone marrow aspiration and biopsy as clinically indicated. In addition, patients undergo standard imaging scans throughout the trial.

After completion of study treatment, patients are followed up three times weekly until day 14, twice weekly from days 15-27, on day 28, at months 2, 3, 6, 9, and 12, every 3 months until the end of the second year, then annually for up to 10 years.

Being tested
Biopsy Procedure, Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Cyclophosphamide
Medical subject headings
Neuroblastoma, Osteosarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biopsy ProcedureProcedure or surgery

    Also called Biopsy, BIOPSY_TYPE, Bx

    Undergo biopsy

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection

    Undergo collection of blood

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow aspiration

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow

    Undergo bone marrow biopsy

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518

    Given IV

  • Echocardiography TestProcedure or surgery

    Also called EC, Echocardiography

    Undergo ECHO

  • Fludarabine PhosphateDrug

    Also called 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara

    Given IV

  • GD2-CAR-expressing Autologous T-lymphocytesBiological

    Also called tvs-CTL Vaccine

    Given IV

  • Imaging ProcedureProcedure or surgery

    Also called Diagnostic Imaging Technique, Image Type, Imaging, Imaging (procedure)

    Undergo standard imaging scans

  • Magnetic Resonance Imaging of the HeartProcedure or surgery

    Also called Cardiac MRI, Heart MRI

    Undergo cardiac MRI

  • Multigated Acquisition ScanProcedure or surgery

    Also called Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan

    Undergo MUGA

  • Questionnaire AdministrationOther

    Ancillary studies

Study groups in NCT04539366
Study groupRoleWhat they receive
Treatment (GD2 CAR T)ExperimentalLYMPHODEPLETION CHEMOTHERAPY: Patients receive fludarabine phosphate IV daily on days -4 to -2 and cyclophosphamide IV daily on days -3 to -2. GD2CART: Patients receive GD2CART cells IV on day 0. Patients also undergo ECHO, MUGA or cardiac MRI scan, blood sample collection throughout the trial, and tumor biopsies and bone marrow aspiration and biopsy as clinically indicated. In addition, patients undergo standard imaging scans throughout the trial.
Receives: Procedure: Biopsy Procedure; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Cyclophosphamide; Procedure: Echocardiography Test; Drug: Fludarabine Phosphate; Biological: GD2-CAR-expressing Autologous T-lymphocytes; Procedure: Imaging Procedure; Procedure: Magnetic Resonance Imaging of the Heart; Procedure: Multigated Acquisition Scan; Other: Questionnaire Administration

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Feasibility of producing GD2-CAR-expressing autologous T-lymphocytes (GD2CART) cells

    Pre-amendment #5: Success will be defined by manufacturing and expansion of GD2CART to satisfy the targeted dose level and meet the requirements of the Certificate of Analysis. Specifically, dose escalation will proceed if 3 or more of the first 3 to 6 patients in a dose level are able to produce adequate cells for evaluation. Amendment #5: Success will be defined by manufacturing and expansion of GD2CART to satisfy the targeted dose level and meet the requirements of the Certificate of Analysis (COA). Specifically, dose escalation will not proceed if less than 3 patients in a dose level are able to produce adequate cells for evaluation.

    Measured: Up to day 28 days after cell infusion

  2. Incidence of adverse events (AEs)

    Safety of GD2CART will be by the incidence and severity of dose limiting toxicities, treatment emergent AEs, serious adverse events, laboratory abnormalities, changes in vital signs, and changes in physical examination following infusion of GD2CART cells.

    Measured: Up to 15 years

  3. Maximum tolerated dose

    Estimate of the maximum tolerated dose of autologous GD2CART in children and young adults with relapsed/refractory osteosarcoma and neuroblastoma following cyclophosphamide-fludarabine based lymphodepletion defined as the dose at which less than 33% of evaluable patients experience a cycle 1 dose limiting toxicity.

    Measured: Up to day 28 days after cell infusion

  4. Best response to GD2CART cells

    Simon's two stage design will be used to evaluate the clinical benefit of GD2CART after conditioning lymphodepletion chemotherapy in two groups of patients: children and young adults with recurrent, refractory osteosarcoma and neuroblastoma.

    Measured: Up to day 28 days after cell infusion

Secondary outcomes (3)

  1. Persistence of GD2CART cells

    Persistence of GD2CART and correlation with tumor regression and sustained clinical benefit will be assessed. Persistence will be defined as the duration of time that GD2CART cells can be detected above the background rate (baseline measure) as measured by polymerase chain reaction. The persistence will be compared between the different response statuses (responders versus non-responders) using a t-test allowing for unequal variance.

    Measured: Up to 5 years

  2. Capacity for rimiducid (AP1903) to reverse unacceptable toxicity related to GD2CART administration

    The toxicity is defined to be reversed or resolved when the toxicity grading has resolved to grade 2 or below. The resolution of toxicity will be summarized descriptively.

    Measured: Up to 28 days after cell infusion

  3. Feasibility and tolerability of a second infusion of GD2CART cells

    The proportion of evaluable patients who produce adequate GD2CART produced that meet Certificate of Analysis (COA) for infusion.

    Measured: After a 2nd infusion of GD2CART cells

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Sep 2020
  2. Study start25 Jan 2022
  3. Primary completion31 Dec 2040 (estimated)
  4. Study completion31 Dec 2040 (estimated)

Why it stopped: Other - review of safety data

Resources

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
73 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04539366
Sponsor's protocol ID
NCI-2020-06646
Other IDs
NCI-2020-06646, PED-CITN-02, PED-CITN-02, U24CA224309, UM1CA154967, UM1CA228823
Status last verified
Aug 2026
First submitted
4 Sep 2020
Last updated
5 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Biopsy Procedure, Biospecimen Collection and Bone Marrow Aspiration in people with Recurrent Childhood Neuroblastoma and Recurrent Childhood Osteosarcoma, run by National Cancer Institute (NCI) at 6 sites 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 45 inclusion and 12 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: paused, as of the sponsor's last update on 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites 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?

Results are not available in the source record.

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?

Feasibility of producing GD2-CAR-expressing autologous T-lymphocytes (GD2CART) cells (time frame: Up to day 28 days after cell infusion). The study lists 4 primary outcomes in all. 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 NCT04539366.

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 5 Aug 2026.

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