Actively Recruiting
A Phase 1/Phase 2, Open-Label Study of Allogeneic Dual-Target GD2/B7-H3 CAR-NK Cells in Children and Young Adults With Relapsed or Refractory Neuroblastoma
Led by Beijing Biotech · Updated on 2026-03-30
36
Participants Needed
1
Research Sites
65 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new treatment using allogeneic dual-target GD2/B7-H3 CAR-NK cells in children and young adults aged 12 months to 21 years with relapsed or refractory neuroblastoma or ganglioneuroblastoma. This Phase 1/Phase 2 study aims to determine the recommended dose and preliminary activity of this engineered cell therapy, which is designed to target specific tumor markers and includes an inducible safety switch to manage side effects. Participants receive a chemotherapy regimen called lymphodepletion with fludarabine and cyclophosphamide before an intravenous infusion of the CAR-NK cells on Day 0. Additional CAR-NK infusions may be given on Days 7 and 14 if safety conditions allow. The study involves a dose-escalation phase to find the optimal dose, followed by an expansion phase to further assess treatment effects in a biomarker-defined group. During the study, participants undergo tumor or marrow testing to confirm target expression and regular disease assessments using revised International Neuroblastoma Response Criteria. Researchers monitor the safety of the treatment by tracking dose-limiting toxicities and adverse events over time. They also study the persistence of CAR-NK cells, immune response markers, and tumor response, with long-term follow-up planned according to regulatory guidelines. Total participation and monitoring may last up to two years or more depending on outcomes and follow-up requirements.
CONDITIONS
Brief Title
Dual-Target GD2/B7-H3 CAR-NK Cells for Pediatric Relapsed or Refractory Neuroblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 12 months to 21 years at consent or assent
- Histologically confirmed neuroblastoma or ganglioneuroblastoma with relapsed, refractory, progressive, or persistent high-risk disease lacking curative standard options
- Measurable or evaluable disease by revised International Neuroblastoma Response Criteria, including MIBG-avid, CT/MRI-evaluable soft-tissue, or bone marrow disease
- Tumor material available for central assessment of GD2 and B7-H3 expression with at least one target positive
- Prior standard neuroblastoma therapy including anti-GD2 therapy unless contraindicated, unavailable, or declined for medical reasons
- Lansky or Karnofsky performance score of 50 or higher
- Life expectancy of at least 8 weeks
- Recovery from acute toxicities of prior therapy and protocol-defined washout periods for chemotherapy, biologics, radiation, and prior cell therapy
- Adequate hematologic, renal, hepatic, cardiac, and pulmonary function per protocol
- Negative pregnancy test for patients of childbearing potential and agreement to use contraception during the protocol period
- Written informed consent from parent/legal guardian and participant assent when appropriate
You will not qualify if you...
- Active uncontrolled infections including bacteremia, uncontrolled viral infection, or invasive fungal disease
- Pregnancy or breastfeeding
- Active grade 2 or higher graft-versus-host disease or systemic immunosuppression for graft-versus-host disease within protocol-defined washout after allogeneic transplant
- Symptomatic or unstable central nervous system disease requiring urgent intervention
- Prior genetically modified cellular therapy within washout window or unresolved toxicity from prior cell therapy
- Active autoimmune disease requiring systemic immunosuppressive therapy
- Clinically significant uncontrolled cardiovascular, pulmonary, hepatic, renal, or neurologic disorders increasing study risk
- Known hypersensitivity to fludarabine, cyclophosphamide, study product components, or required supportive medications
- Known uncontrolled HIV infection or uncontrolled hepatitis B or C
- Any medical, psychosocial, or logistical condition judged by investigators to impair safety or protocol adherence
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 14 days
Participants receive lymphodepleting chemotherapy followed by an infusion of allogeneic dual-target GD2/B7-H3 CAR-NK cells on Day 0. Additional infusions may occur on Days 7 and 14 if safety criteria are met and there is no rapid disease progression.
3 infusion visits (in-person)
Duration - Up to 24 months
Participants are monitored for safety, treatment response, and long-term outcomes including adverse events and disease progression for up to 24 months after treatment.
Regular visits during the first 12 months and periodic visits up to 24 months
Trial Site Locations
Total: 1 location
1
Peking University Shenzhen Hospital
Shenzhen, Guangdong, China, 518036
Actively Recruiting
Research Team
S
Seni S Lu, Phd
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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