ClinicalTrials.gov record NCT04637503

4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 5, 2026

Multi-center Phase I/II Clinical Trial of 4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
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 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).
Age1 year old to 65 years old
SexAll
LocationChina
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.

This Phase I/II interventional study evaluates 4SCAR-T cell therapy that targets GD2, PSMA and CD276 in people with relapsed or refractory neuroblastoma. Tumor tissue is tested by immunohistochemistry to identify which of these antigens are highly expressed; two or three targets are combined for each patient's CAR-T product. Participants receive an intravenous infusion of gene-modified T cells (1x10^6–1x10^7 cells/kg) and the study tracks safety, tumor response, CAR-T expansion/persistence, cytokine profiles and survival.

What it is studying. The study is testing the feasibility, safety, and anti-tumor activity of a multi-antigen 4SCAR-T cell infusion for relapsed or refractory neuroblastoma, and it is studying how these engineered T cells function and persist in patients.

What the researchers hope to learn. Scientifically, the study aims to determine the safety and toxicity profile of combined GD2/PSMA/CD276 4SCAR-T cells (using CTCAE v4.0), measure anti-tumor responses (including complete and very good partial responses), characterize CAR-T cell expansion and persistence in blood, describe cytokine secretion patterns after infusion, and record overall survival times.

What this study is about

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

The purpose of this clinical trial is to assess the feasibility, safety and efficacy of multiple 4SCAR-T cell therapy which targets GD2, PSMA and CD276 surface antigens in patients with relapsed and refractory neuroblastoma (NB). Another goal of the study is to understand the function of the multi-CAR-T cells and their persistency in the patients.

Interventions being studied

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

Biological
  • GD2, PSMA and CD276 CAR-T cells

Eligibility

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

Inclusion criteria

  • Patients with tumors have received standard first-line therapy and been judged to be non-resectable, metastatic, progressive or recurrent.
  • The expression status of GD2, PSMA and CD276 antigens of the tumor will be determined for eligibility. Positive expression is defined by GD2, PMSA and CD276 antibody staining results based on immunohistochemistry or flow cytometry analyses.
  • Body weight greater than or equal to 10 kg.
  • Age: ≥1 year and ≤ 65 years of age at the time of enrollment.
  • Life expectancy: at least 8 weeks.
  • Prior Therapy:
  • There is no limit to the number of prior treatment regimens. Any grade 3 or 4 non-hematologic toxicity of any previous therapy must have resolved to grade 2 or less.
  • Participant must not have received hematopoietic growth factors for at least 1 week prior to mononuclear cells collection.
  • At least 7 days must have elapsed since the completion of therapy with a biologic agent, targeted agent, tyrosine kinase inhibitor or a metronomic non-myelosuppressive regimen.
  • At least 4 weeks must have elapsed since prior therapy that included a monoclonal antibody.
  • At least 1 week since any radiation therapy at the time of study entry.
  • Karnofsky/jansky score of 60% or greater.
  • Cardiac function: Left ventricular ejection fraction greater than or equal to 40/55 percent.
  • Pulse Ox greater than or equal to 90% on room air.
  • Liver function: defined as alanine transaminase (ALT) <3x upper limit of normal (ULN), aspartate aminotransferase (AST) <3x ULN; serum bilirubin and alkaline phosphatase <2x ULN.
  • Renal function: Patients must have serum creatinine less than 3 times upper limit of normal.
  • Marrow function: White blood cell count ≥1000/ul, Absolute neutrophil count ≥500/ul, Absolute lymphocyte count ≥500/ul, Platelet count ≥25,000/ul (not achieved by transfusion).
  • Patients with known bone marrow metastatic disease will be eligible for study as long as they meet hematologic function criteria, and the marrow disease not evaluable for hematologic toxicity.
  • For all patients enrolled in this study, their parents or legal guardians must sign an informed consent and assent.

Exclusion criteria

  • Existing severe illness (e.g. significant cardiac, pulmonary, hepatic diseases, etc.) or major organ dysfunction, with the exception of grade 3 hematologic toxicity.
  • Untreated central nervous system (CNS) metastasis: Patients with previous CNS tumor involvement that has been treated and is stable for at least 6 weeks following completion of therapy are eligible.
  • Previous treatment with other genetically engineered GD2, PSMA and CD276 CART cells.
  • Active HIV, Hepatitis B virus (HBV), Hepatitis C virus (HCV) infection or uncontrolled infection.
  • Patients who require systemic corticosteroid or other immunosuppressive therapy.
  • Evidence of tumor potentially causing airway obstruction.
  • Inability to comply with protocol requirements.
  • Insufficient CART cells availability.

Study description and what participation involves

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

Show the detailed description from the registry

Important Regulatory Notice: This trial record is only for global academic information registration on ClinicalTrials.gov. Neither the sponsor Beijing Meikang Jimian Biotechnology Co., Ltd. nor collaborator Shenzhen Geno-Immune Medical Institute has obtained NMPA clinical trial approval or clinical technology filing permission to carry out interventional cell therapy trials in mainland China. ClinicalTrials.gov registration alone does not represent legal approval by Chinese health and drug regulatory authorities. Neuroblastoma is one of the most aggressive childhood tumors arising from neural crest cells. Nearly 50% of patients with high risk disease have poor long-term survival even after multimodal treatments. Novel immunotherapy targeting tumor-specific antigens has been developed to meet the desperate need. Disialoganglioside (GD2) is a well-studied tumor associated antigen which is expressed uniformly in NB but restrictedly in normal tissue. Over the past few years, CAR-T therapy against GD2 in NB has achieved encouraging but modest outcomes. Only a fraction of patients achieved measurable responses. Like for many other solid tumors, CAR-T therapy for NB is not as effective as for hematologic malignancies. In this study, the investigators use "multiple targeting" approach as the strategy to overcome the challenge in treating NB. Prostate-specific membrane antigen (PSMA) is expressed in normal prostate but is upregulated in prostate tumor. However, PSMA expression is not restricted to prostate cancer. By immunohistochemistry (IHC) staining, the investigators confirmed that PSMA is expressed in a variety of solid tumors, including brain tumor, neuroblastoma and some lymphoma tumor tissues. Therefore, PSMA could potentially serve as a promising target for antigen-specific immunotherapy in patients with NB. In addition, CD276 (B7-H3) is an immune checkpoint molecule highly expressed on many solid tumors including NB. CD276 has been characterized to be involved in tumor evasion and thus its expression is correlated with poor prognosis. These characteristics make CD276 an attractive candidate for immunotherapy. Given the significant variation of tumor antigen expression among patients, the investigators aim to examine GD2, PSMA and CD276 expression in each patient's tumor sections by IHC staining, and combine two or three highly-expressed targets for the 4SCAR-T therapy. This individualized and multi-antigen-targeted approach is a new strategy to overcome the limited clinical outcome in the 4SCAR-T therapy against NB. The purpose of this clinical study is to assess the feasibility, safety and efficacy of the combinational GD2, PSMA and CD276 4SCAR-T cell therapy against NB. Another goal of the study is to learn more about the function of the multi-4SCAR-T cells and their persistency in the patients.

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.

Your tumor will be tested for three surface proteins (GD2, PSMA and CD276) using immunohistochemistry or flow cytometry. Investigators will collect your mononuclear cells to manufacture gene-modified CAR‑T cells, and you will receive an intravenous (IV) infusion of those GD2/PSMA/CD276 CAR‑T cells (1×10^6–1×10^7 cells/kg noted in the record). After infusion you will return for regular follow-up over months to years that includes blood draws to monitor CAR‑T cell levels and cytokine profiles, imaging scans to assess tumor response, and monitoring for side effects and overall survival.

How the study is structured. An interventional, single-group (no masking) Phase I/II trial. Eligible participants receive an IV infusion of GD2/PSMA/CD276 4SCAR-T cells. Investigators will monitor participants for adverse events, assess tumor response by imaging, and take blood tests to measure CAR-T cell expansion, persistence and cytokine secretion over follow-up.

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 registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in China. 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?

More on Gaucher disease

Clinical trials64 trials listed for Gaucher diseaseEvery trial the registry lists for Gaucher disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Gaucher disease researchLatest monthly update: September 2026. Includes: Platelet release. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 31, 2026 (estimated)
Primary completion
Dec 31, 2029 (estimated)
Study completion
Dec 31, 2030 (estimated)
Target enrollment
100 participants (estimated)
Last updated on registry
Sep 1, 2026
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
Shenzhen Geno-Immune Medical Institute

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

relapsed
A condition that improved with treatment and has since returned or worsened.
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.
CAR-T cells
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.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
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.
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.
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.
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.
Absolute neutrophil count
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.
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.
corticosteroid
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.
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.
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.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.

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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