Real-World Outcomes of First-Line Treatment of Metastatic or Locally Advanced Upper Tract Urothelial Carcinoma With Enfortumab Vedotin Plus Pembrolizumab : A French Multicenter Retrospective Observational Study

NCT07633353 | Enrolling By Invitation FDA Drug

• 1 other identifier: LOCAL/2026/CG-01
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

The combination of Enfortumab Vedotin and Pembrolizumab has substantially improved outcomes for patients with advanced urothelial carcinoma and has emerged as a new standard of care in the first-line setting. However, data specifically regarding its use in patients with Upper Tract Urothelial Carcinoma (UTUC) remain limited. This multicenter retrospective observational study aims to evaluate the real-world efficacy and safety of Enfortumab Vedotin plus Pembrolizumab as first-line treatment in patients with metastatic or locally advanced Upper Tract Urothelial Carcinoma (UTUC). The primary outcome will include objective response rate (ORR). Secondary outcomes will include progression-free survival (PFS), overall survival (OS), time to treatment response (TTR) and treatment-related toxicity. In addition, the study will explore potential associations between clinical outcomes and selected molecular and immunohistochemical biomarkers, including Nectin-4 expression, microsatellite instability (MSI) status, and programmed death-ligand 1 (PD-L1) expression. These exploratory analyses may help identify predictive factors of response and contribute to a better understanding of treatment outcomes in this specific patient population.

Conditions

Advanced Upper Tract Urothelial Carcinoma

Keywords

enfortumab-vedotin; pembrolizumab

Outcome Measures

Primary Outcomes (1)

• objective response rate

  number of patients presenting with treatment response from the beginning of the treatment to at least 6 months follow up

Interventions

enfortumab vedotin plus pembrolizumab in first line setting for advanced upper tract urothelial carcinoma DRUG

enfortumab vedotin plus pembrolizumab for advanced upper tract urothelial carcinoma, from september 2024 (time of the French ATU) to december 2025 (time of inclusion minus 6 months follow up)

Eligibility Criteria

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

Study Population

adults \> 18 years old with histologically confirmed metastatic or locally advanced urothelial carcinoma

You may qualify if:

• advanced upper tract urothelial carcinoma treated with enfortumab vedotin and pembrolizumab
• at least one infusion of the treatment

You may not qualify if:

• patient previously treated with immune checkpoint inhibitor
• patient previously treated with systemic treatment for advance UTUC

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Nīmes: lead

Study Sites (1)

Centre Hospitalier Universitaire

Nîmes, 30000, France

Location

Related Publications (1)

• Powles T, Valderrama BP, Gupta S, Bedke J, Kikuchi E, Hoffman-Censits J, Iyer G, Vulsteke C, Park SH, Shin SJ, Castellano D, Fornarini G, Li JR, Gumus M, Mar N, Loriot Y, Flechon A, Duran I, Drakaki A, Narayanan S, Yu X, Gorla S, Homet Moreno B, van der Heijden MS; EV-302 Trial Investigators. Enfortumab Vedotin and Pembrolizumab in Untreated Advanced Urothelial Cancer. N Engl J Med. 2024 Mar 7;390(10):875-888. doi: 10.1056/NEJMoa2312117.

  PMID: 38446675 BACKGROUND

Biospecimen

Retention: SAMPLES WITHOUT DNA

tissue from available biopsies will be retained, no additionnal samples will be retained

MeSH Terms

Interventions

enfortumab vedotin; pembrolizumab

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Intern

Study Record Dates

• First Submitted: June 3, 2026
• First Posted: June 8, 2026
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: June 8, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)