Phase 12 Study of Dual-Target GD2B7-H3 CAR-NK Cell Therapy in Children and Young Adults With Relapsed or Refractory Neuroblastoma

What this Trial Is About

Researchers are evaluating a new treatment using allogeneic dual-target GD2B7-H3 CAR-NK cells in children and young adults aged 12 months to 21 years with relapsed or refractory neuroblastoma or ganglioneuroblastoma. This Phase 1Phase 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.

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