224P - WUTSUP-01: Phase II trial of neoadjuvant toripalimab and chemotherapy in locally advanced upper tract urothelial carcinoma

Background

For locally advanced upper tract urothelial carcinoma (UTUC), no evidence proved the effectiveness and safety of neoadjuvant immunotherapy combined with chemotherapy. Toripalimab is an innovative anti-PD-1 monoclonal antibody approved in China across multiple cancer indications. The current trial aims to investigate the effectiveness and safety of neoadjuvant toripalimab combined with chemotherapy in patients with locally advanced UTUC.

Methods

This is a single-arm, phase 2 prospective trial. This trial aims to recruit 59 patients aged 18-75 years with cT2-4aN0-2M0 UTUC. Patients receive toripalimab (240mg p.o.) plus gemcitabine (1000mg/m² on D1 and D8) and cisplatin (75mg/m², D1) every 3 weeks for 3-4 cycles before radical nephroureterectomy (RU). Tumor response is determined according to RECIST 1.1 criteria. The primary outcome is pathologic complete response (pCR), which was defined as the absence of invasive cancer or in situ disease in the kidney and ureter after RU (ypT0N0M0).

Results

11 patients have been recruited in this trial. 9 patients had completed neoadjuvant combination therapy and successfully underwent RU. 3 out of the 9 patients (33.3%) achieved pCR after neoadjuvant combination therapy and 4 out of the 9 patients (44.4%) attained pathologic partial response. No treatment-related adverse event was observed among all patients.

Conclusions

Neoadjuvant toripalimab combined with chemotherapy appears be effective and well-tolerated for patients with locally advanced UTUC. This trial is continuing to recruit patients.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

Y. Bao.

Funding

Shanghai Junshi Biosciences Co., Ltd.

Disclosure

All authors have declared no conflicts of interest.