(UroToday.com) The 2026 American Society of Clinical Oncology Genitourinary (ASCO GU) cancers symposium held in San Francisco, CA, was host to the Trials in Progress Poster Session B: Urothelial Carcinoma. Dr. Yige Bao presented the trial in progress: A phase II study evaluating the efficacy and safety of perioperative EV + toripalimab combined with endoscope-based kidney-sparing surgery for patients with high-risk UTUC (WUTSUP-04EV-JS001).
Dr. Bao began by highlighting a key clinical dilemma: radical nephroureterectomy remains the standard for high-risk UTUC but frequently compromises renal function, limiting eligibility for adjuvant therapy. Conversely, kidney-sparing surgery carries substantial recurrence risk. Given the established activity of enfortumab vedotin combined with PD-1 inhibition in advanced urothelial carcinoma,1 this trial evaluates whether systemic therapy (EV + toripalimab) can downstage tumors, enable successful kidney-sparing surgery, and preserve long-term oncologic control.
WUTSUP-04 is a prospective, single-center phase II study conducted at West China Hospital, Sichuan University (CTEC: 2025-763). Eligible patients are adults with histologically confirmed high-risk, non-metastatic UTUC staged cTa-2N0M0, with tumor size ≤20 mm or disease deemed endoscopically resectable. Patients must also have an imperative or relative indication for nephron preservation, such as solitary kidney, bilateral UTUC, chronic kidney disease, or a strong preference for kidney-sparing surgery after informed consent.
In parallel, data will be prospectively collected from contemporaneous patients, meeting similar eligibility criteria, who undergo radical nephroureterectomy by the same lead surgeon, allowing comparison of long-term oncologic outcomes between strategies.
Treatment is delivered sequentially. During the induction phase, patients receive three cycles of enfortumab vedotin at 1.25 mg/kg intravenously on days 1 and 8, combined with toripalimab 240 mg intravenously on day 1, administered every three weeks. After response assessment and local tumor ablation, patients proceed to a consolidation phase consisting of six additional cycles of enfortumab vedotin plus toripalimab, followed by toripalimab monotherapy to complete one year of total systemic therapy.
The primary endpoint is conversion-free survival, defined as the time from enrollment to radical nephroureterectomy performed for any UTUC-related indication or death. Secondary endpoints include clinical complete response rate after induction, disease-free survival, metastasis-free survival, overall survival, renal function preservation, and safety.
The study includes an initial safety run-in of six patients. A total of 55 participants are planned to estimate the 12-month conversion-free survival rate with a 95 percent confidence interval width of approximately 0.087 under an exponential survival model.
Lastly, Dr. Bao noted that this trial has received ethics approval and is currently initiating patient enrollment.
Presented by: Yige Bao, M.D., Ph.D., Oncological Urologist and Microbiome researcher in West China Hospital, Sichuan University
Written by: Julian Chavarriaga, MD – Urologic Oncologist, Department of Urology at Penn State Health. @chavarriagaj on Twitter during the 2026 American Society of Clinical Oncology Genitourinary (ASCO GU) cancers symposium held in San Francisco, CA, between February 26th and 28th, 2026.
References:- Powles T, Rosenberg JE, Sonpavde GP, Loriot Y, Durán I, Lee JL, Matsubara N, Vulsteke C, Castellano D, Wu C, Campbell M, Matsangou M, Petrylak DP. Enfortumab Vedotin in Previously Treated Advanced Urothelial Carcinoma. N Engl J Med. 2021 Mar 25;384(12):1125-1135. doi: 10.1056/NEJMoa2035807. Epub 2021 Feb 12. PMID: 33577729; PMCID: PMC8450892.