Neoadjuvant Chemotherapy Before Surgery vs Surgery Alone for UTUC: A Randomized Trial

NCT07492225 | Active Not Recruiting Phase 2

• 1 other identifier: CH-UTUC NAC vs NNAC
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

This clinical trial aims to compare the effectiveness and safety of two treatment options for patients with high-risk upper tract urothelial carcinoma (UTUC). One group will receive neoadjuvant chemotherapy with gemcitabine and cisplatin followed by surgery (radical nephroureterectomy), while the other group will undergo surgery without chemotherapy. The primary goal is to assess the pathological response to treatment, focusing on how much the tumor shrinks before surgery. The trial will also evaluate the safety of the treatments, overall survival, and recurrence-free survival. This study will help determine whether adding chemotherapy before surgery improves outcomes for patients with high-risk UTUC.

Conditions

Neoadjuvant Therapy; Urothelial Carcinoma Ureter; Upper Urinary Tract Urothelial Carcinoma

Outcome Measures

Primary Outcomes (1)

• Pathological Response Rate

  Proportion of patients with ypT1N0 or lower after surgery in the neoadjuvant chemotherapy + partial nephrectomy group compared to the direct partial nephrectomy group.

  6 weeks after surgery

Secondary Outcomes (4)

• Chemotherapy Safety and Tolerability

  During chemotherapy and post-surgery follow-up

• Overall Survival (OS)

  24 months after surgery

• Cancer-Specific Survival (CSS)

  24 months after surgery

• Recurrence-Free Survival (RFS)

  24 months after surgery

Study Arms (2)

NAC+RNU

EXPERIMENTAL

Drug: Neoadjuvant Gemcitabine + Cisplatin Chemotherapy

RNU

NO INTERVENTION

Interventions

Neoadjuvant Gemcitabine + Cisplatin Chemotherapy DRUG

Patients in the experimental group will receive neoadjuvant chemotherapy with a combination of gemcitabine and cisplatin before undergoing surgery. The chemotherapy regimen consists of: Gemcitabine: 1000 mg/m², administered on Day 1 and Day 8 of each 21-day cycle. Cisplatin: 70 mg/m², administered on Day 2 of each 21-day cycle. This treatment will be repeated for 3 to 4 cycles. The goal of this chemotherapy is to shrink the tumor and improve the chances of a successful surgery. After completing the chemotherapy, patients will undergo radical nephroureterectomy (RNU), a surgery to remove the kidney and ureter affected by cancer, within 6 weeks (+/- 2 weeks) of the last chemotherapy cycle. This approach is designed to evaluate if chemotherapy before surgery can improve pathological response and long-term survival outcomes.

NAC+RNU

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically confirmed high-risk upper tract urothelial carcinoma (UTUC).
• Clinically non-metastatic UTUC (N≤1, M0) based on imaging (CT/MRI).
• Planned for radical nephroureterectomy (RNU).
• ECOG performance status of 0 or 1.
• Sufficient renal function (GFR ≥45 mL/min) and able to tolerate chemotherapy with cisplatin.
• Age ≥18 years.
• Informed consent signed.

You may not qualify if:

• Imaging shows ≥N2 disease or metastatic disease (M1).
• History of invasive or lymph node-positive UTUC or invasive disease in the contralateral upper tract in the last 2 years.
• Single kidney or contraindication to cisplatin.
• Currently participating in other interventional clinical trials.
• History of non-urothelial malignancies (except treated skin basal cell carcinoma or cervical carcinoma in situ).
• Pregnant or breastfeeding women.
• Any other medical condition deemed inappropriate by the treating physician.

Sponsors & Collaborators

• Changhai Hospital: lead

Study Sites (1)

Changhai Hospital

Shanghai, Shanghai Municipality, 200090, China

Location

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2026
• First Posted: March 25, 2026
• Study Start: August 15, 2025
• Primary Completion (Estimated): August 15, 2027
• Study Completion (Estimated): August 15, 2029
• Last Updated: March 25, 2026

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)