NEO-ERA-01 Trial

Total planned enrollment is 30 patients, accounting for a 10% dropout rate. A single-arm design is justified because no established neoadjuvant standard exists for ICC. The trial was initiated in December 2023 and is expected to complete by end of 2026. The trial will disseminate findings through peer-reviewed publications regardless of whether the hypothesis is confirmed. The primary endpoint is the proportion of patients who complete at least two neoadjuvant cycles and then undergo curative-intent resection. NEO-ERA-01 is a prospective, multicentre, single-arm phase II feasibility trial conducted in China using Simon's two-stage design. The Stage 1 interim analysis is planned approximately 8 months after the first patient is enrolled, or when 12 patients complete neoadjuvant evaluation. Exploratory analyses in NEO-ERA-01 aim to identify biomarkers that predict benefit from neoadjuvant immunotherapy in ICC.