2026-09-16 15:06 |
[DKFZ-2026-02290]
Journal Article
Pinto, N. ; Künkele, A. ; Albert, C. M. ; et al
The First-in-Human ENCIT01 Trial Comparing Second- versus Third-Generation L1CAM-specific CAR T Cells in Patients with Primary Refractory or Relapsed Neuroblastoma.
Outcomes for children with relapsed and refractory neuroblastoma are dismal. ENCIT-01 (NCT02311621) was a first-in-human clinical trial for patients with relapsed and refractory neuroblastoma using chimeric antigen receptor (CAR) T cells targeting L1CAM, an adhesion molecule that is overexpressed in neuroblastoma with limited normal tissue expression.This trial evaluated three different CAR constructs: a short-spacer second-generation 4-1BB CAR (2GS, arm A), a short-spacer third-generation 4-1BB + CD28 CAR (3GS, arm B), and a long-spacer second-generation 4-1BB CAR (2GL, arm C).Thirty-six patients were enrolled, of whom 22 were treated (arm A/2GS n = 11, arm B/3GS n = 8, and arm C/2GL n = 3). [...]
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