The First-in-Human ENCIT01 Trial Comparing Second- versus Third-Generation L1CAM-specific CAR T Cells in Patients with Primary Refractory or Relapsed Neuroblastoma.
Clin Cancer ResSep 15, 2026
Clinical ResearchOncology (Medical)
Navin Pinto, Annette Künkele, Catherine M Albert et al.
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In 30 seconds
The ENCIT-01 trial was a Phase I clinical study evaluating L1CAM-specific CAR T cells in 36 children with relapsed or refractory neuroblastoma. The trial compared second- and third-generation CAR constructs. No objective responses were observed, and common toxicities included cytokine release syndrome and hyponatremia, with the latter being dose-limiting in three patients.
Key findings
- No objective responses were observed in any of the treatment arms.
- Cytokine release syndrome, skin rash, and hyponatremia were common ≥ grade 2 toxicities.
- Hyponatremia was dose-limiting in three patients across different treatment arms.
- Patterns of toxicity appeared at lower dose levels in third-generation and long-spacer CAR arms.
Why it matters
The study highlights the challenges in treating relapsed neuroblastoma with CAR T cell therapy, particularly regarding the safety and efficacy of targeting L1CAM. Understanding these limitations is crucial for future therapeutic strategies.