Testing modified immune cells in children with hard-to-treat neuroblastoma

Trial details

Who runs it

Beijing Biotech

Trial code

NCT07502287

Record updated

30 March 2026

Original description

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 activityRead full original descriptionShow less

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

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

Inclusion: 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: 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; Prior genetically modified cellular therapy within the protocol-defined washout window, or unresolved clinically significant toxicity from prior cell therapy; Active uncontrolled infection, including bacteremia, uncontrolled viral infection, or invasive fungal disease; Symptomatic or unstable central nervous system disease requiring urgent medical intervention; 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; Pregnancy or breastfeeding.

ClinicalTrials.gov NCT07502287