NCT06774131

A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer

A Phase 3, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of UGN-104, a Novel Formulation of UGN-101, for the Treatment of Patients With Low-grade Upper Tract Urothelial Cancer (LG-UTUC)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 70 (estimated)
• Sponsor: UroGen Pharma Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the efficacy and safety of UGN-104, a new formulation of UGN-101 (approved in the United States and Israel as JELMYTO \[mitomycin\] for pyelocalyceal solution), instilled in the upper urinary tract (UUT) of patients with low-grade upper tract urothelial cancer (LG-UTUC).

Detailed description

Eligible patients will be treated with UGN-104 once weekly for 6 weeks (a total of 6 doses). Efficacy will be assessed by the complete response rate (CRR) at the Primary Disease Evaluation (PDE) Visit (approximately 3 months after the first instillation). Response will be determined based on central laboratory interpretation of UUT wash urine cytology and visual evaluation (ureteroscopy). Biopsy of any remaining or new lesions will be performed where technically feasible and the central laboratory histopathology assessment will also be used in the response determination, if applicable. Patients who have a complete response (CR) at the PDE Visit, defined as having no detectable disease (NDD), will enter the Follow-up Period of the study. During the Follow-up Period, patients may receive UGN-104 as maintenance treatment at the discretion of the investigator. The maintenance regimen is once monthly for 11 months (a total of 11 doses) and should start as soon as possible (no longer than 3 weeks) after CR is determined at the PDE Visit. All patients in the Follow-up Period, regardless of whether they receive maintenance treatment, will return to the clinic every 3 months for evaluation of response and will remain on study for 12 months after the PDE Visit or until disease recurrence, disease progression, or death, whichever occurs first. Patients who have a non-complete response (NCR) at the PDE Visit will be considered to have completed the study.

Conditions

• Upper Urinary Tract Urothelial Carcinoma
• Urothelial Carcinoma

Interventions

Type	Name	Description
DRUG	UGN-104	UGN-104 consists of mitomycin and sterile hydrogel (a proprietary thermally responsive gel) that is used to reconstitute mitomycin before instillation. The reverse thermal properties of UGN-104 allow for local administration of mitomycin as a liquid under chilled conditions, with subsequent conversion to a semi-solid gel depot following instillation into the UUT.

Timeline

• Start date: 2025-08-06
• Primary completion: 2026-10-01
• Completion: 2027-10-01
• First posted: 2025-01-14
• Last updated: 2026-01-16

Locations

42 sites across 7 countries: United States, Bulgaria, Georgia, Israel, Poland, Romania, Spain

Regulatory

• FDA-regulated drug study