ClinicalTrials.gov record NCT07502287

Dual-Target GD2/B7-H3 CAR-NK Cells for Pediatric Relapsed or Refractory Neuroblastoma

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

A Phase 1/Phase 2, Open-Label Study of BiomarkerInformed, Allogeneic Dual-Target GD2/B7-H3 (CD276) CAR-NK Cells in Children and Young Adults With Relapsed or Refractory 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 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 21 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 1/Phase 2 interventional study evaluates cord blood–derived allogeneic NK cells engineered to target GD2 and B7-H3 in children and young people with relapsed or refractory neuroblastoma. Part A uses a 3+3 dose-escalation to define the recommended Phase 2 dose (RP2D); Part B expands at the RP2D to estimate preliminary activity in a biomarker-characterized group. Participants receive protocol lymphodepletion followed by an IV CAR-NK infusion on Day 0, with optional additional infusions on Days 7 and 14 if safety criteria are met. Tumor or marrow tissue must be tested centrally for GD2 and B7-H3 expression before treatment.

What it is studying. The study is testing the safety, tolerability, and dosing (to find an RP2D) and the preliminary anti-tumor activity of allogeneic dual-target GD2/B7-H3 CAR-NK cells, plus how CAR-NK expansion/persistence and tumor antigen levels relate to response.

What the researchers hope to learn. Determine dose-limiting toxicities and a recommended Phase 2 dose; characterize treatment-emergent adverse events including CRS and ICANS; estimate objective response rate, duration of response, progression-free survival, and overall survival; assess CAR-NK expansion and persistence, cytokine kinetics, and the relationship between tumor antigen density and response to inform future development strategies (dual-target versus GD2- or B7-H3-focused).

What this study is about

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

This illustrative Phase 1/Phase 2 study tests allogeneic dual-target GD2/B7-H3 (CD276) CAR-NK cells in children and young adults with relapsed or refractory neuroblastoma. After lymphodepletion, participants receive IV CAR-NK cells;Part A defines the RP2D and Part B estimates preliminary activity

Interventions being studied

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

Drug
  • Fludarabine
  • Cyclophosphamide
Biological
  • EB-DTNB-NK

Eligibility

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

Inclusion criteria

  • Age 12 months to 21 years at consent/assent.
  • Histologically confirmed neuroblastoma or ganglioneuroblastoma with relapsed, refractory, progressive, or persistent high-risk disease for which no curative standard option is available.
  • Measurable or evaluable disease according to revised International Neuroblastoma Response Criteria (rINRC), including MIBG-avid disease, CT/MRI-evaluable soft-tissue disease, and/or bone marrow disease.
  • Tumor material available for central assessment of GD2 and B7-H3 expression; at least one target must be positive.
  • GD2 is treated as the core target and B7-H3 as the complementary target for correlative target-prioritization analyses.
  • Prior exposure to standard neuroblastoma therapy, including anti-GD2-based therapy, unless contraindicated, unavailable, or declined for a documented medical reason.
  • Lansky or Karnofsky performance score >= 50.
  • Life expectancy >= 8 weeks.
  • Recovery from clinically significant acute toxicities of prior therapy and protocol-defined washout from chemotherapy, biologics, radiation, and prior cell therapy.
  • Adequate organ function: hematologic, renal, hepatic, cardiac, and pulmonary function considered sufficient by protocoldefined laboratory and clinical thresholds.
  • Negative pregnancy test for patients of childbearing potential and agreement to use effective contraception during the protocol-defined period.
  • Written informed consent from parent/legal guardian and assent from the participant when appropriate.

Exclusion criteria

  • Active uncontrolled infection, including bacteremia, uncontrolled viral infection, or invasive fungal disease.
  • Pregnancy or breastfeeding.
  • Active grade >= 2 graft-versus-host disease, or systemic immunosuppression for treatment/prevention of graft-versushost disease within the protocol-defined washout period after prior allogeneic transplant.
  • Symptomatic or unstable central nervous system disease requiring urgent medical intervention.
  • Prior genetically modified cellular therapy within the protocol-defined washout window, or unresolved clinically significant toxicity from prior cell therapy.
  • Active autoimmune disease requiring systemic immunosuppressive therapy.
  • Clinically significant uncontrolled cardiovascular, pulmonary, hepatic, renal, or neurologic disorder that would increase study risk.
  • Known hypersensitivity to fludarabine, cyclophosphamide, study-product components, or supportive-care medications required by the protocol.
  • Known uncontrolled HIV infection or uncontrolled hepatitis B or C.
  • Any medical, psychosocial, or logistical condition that, in the investigator's judgment, would make study participation unsafe or would impair protocol adherence.

Study description and what participation involves

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

Detailed description from the registry

The investigational product in this example is a cord blood-derived allogeneic NK-cell therapy engineered to express a dual-target CAR recognizing GD2 and B7-H3, supported by IL-15 to improve short-term persistence and equipped with an inducible safety switch. The study is designed as a multicenter Phase 1/Phase 2 protocol: Part A uses a standard 3+3 dose-escalation approach across predefined dose levels, and Part B expands at the RP2D in a biomarker-characterized population. All participants undergo central review of tumor tissue or marrow for GD2 and B7-H3 expression before treatment. Patients receive protocol-defined lymphodepletion followed by CAR-NK infusion on Day 0, with optional additional infusions on Days 7 and 14 if there is no dose-limiting toxicity (DLT), uncontrolled cytokine release syndrome (CRS), or rapid progression. Formal disease assessment uses revised International Neuroblastoma Response Criteria (rINRC). Correlative studies assess CAR-NK expansion and persistence, cytokine kinetics, tumor-response associations with antigen density, and whether future development should remain dualtarget or shift toward a GD2-dominant or B7-H3-enriched strategy. Long-term follow-up for gene-modified cellular therapy is planned per local regulatory requirements.

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.

Before treatment, tumor or marrow samples are sent for central testing of GD2 and B7‑H3. Participants receive protocol‑defined lymphodepleting chemotherapy (fludarabine and cyclophosphamide) followed by an intravenous infusion of the allogeneic dual‑target CAR‑NK cells on Day 0, with optional additional infusions on Days 7 and 14 if safety criteria are met. Expect close monitoring for side effects (including cytokine release syndrome and neurologic events), blood samples for correlative immune and persistence studies, and formal disease assessments (imaging and marrow checks) using revised International Neuroblastoma Response Criteria. Long‑term follow‑up for gene‑modified cellular therapy is planned per local regulatory requirements.

How the study is structured. Open-label, single-group interventional study with Phase 1 dose-escalation (standard 3+3) followed by a Phase 2 expansion at the RP2D. Participants receive protocol-defined fludarabine/cyclophosphamide lymphodepletion, an IV CAR-NK infusion on Day 0, and optional repeat infusions on Days 7 and 14 if there is no prohibitive toxicity or rapid progression. Central testing of tumor or marrow for GD2 and B7-H3 is performed before treatment. Correlative studies and long-term follow-up for gene-modified cell therapy are included.

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 Relapsed Neuroblastoma; Refractory Neuroblastoma; High-Risk Neuroblastoma; Ganglioneuroblastoma. 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 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
Mar 2, 2026
Primary completion
Mar 14, 2027 (estimated)
Study completion
Jun 17, 2028 (estimated)
Target enrollment
36 participants (estimated)
Last updated on registry
Mar 30, 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
Beijing Biotech

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

Phase 1
An 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.
Phase 2 study
A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
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.
RP2D
The dose chosen at the end of a phase 1 study for testing in later studies, based on safety and other measurements.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
Histologically confirmed
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.
relapsed
A condition that improved with treatment and has since returned or worsened.
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.
MRI
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.
contraindicated
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
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.
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.
washout
A period during which a participant stops a previous treatment before starting the study treatment, so the earlier treatment does not affect the results.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Written 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.
legal guardian
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
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.
investigational product
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.
3+3
Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
biomarker
Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
dose-limiting toxicity
A side effect, defined in advance, that is severe enough to limit how far the dose can be raised in a dose-escalation study. How many such events stop escalation is set by the study's rules.
cytokine release syndrome
A reaction in which the immune system releases a large amount of signalling proteins at once, causing fever and sometimes low blood pressure or breathing problems. It can occur after some immune therapies, such as CAR-T cells or bispecific antibodies.
Long-term follow-up
Observation that continues for years after the study treatment, often required for gene and cell therapies.

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.

← Back to clinical trials