NCT05375903

A Phase 1 Dose-escalation Study of UGN-301 in Patients With Recurrent Non-muscle Invasive Bladder Cancer (NMIBC)

A Phase 1, Open-label, Dose-escalation Study to Investigate the Safety, Tolerability, and Pharmacokinetics of UGN-301 (Zalifrelimab) Administered Intravesically as Monotherapy and in Combination With Other Agents in Patients With Recurrent NMIBC

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 51 (actual)
• Sponsor: UroGen Pharma Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is being conducted to evaluate the safety and determine the recommended Phase 2 dose (RP2D) of UGN-301 (zalifrelimab) administered intravesically as monotherapy and in combination with other agents in patients with recurrent NMIBC.

Detailed description

This master protocol will comprise multiple treatment arms designed to independently investigate intravesical delivery of UGN-301 either as monotherapy or in combination with other agents. Initial study treatment arms will include: * UGN-301 monotherapy * UGN-301 + UGN-201 (imiquimod) in combination * UGN-301 + gemcitabine in combination Additional study treatment arms investigating UGN-301 in combination with other agents may be added in the future. The study will evaluate escalating doses of UGN-301 to determine the biologically effective dose (BED) and maximum tolerated dose (MTD) of UGN-301 either as monotherapy or in combination with other agents. When evaluated in combination with other agents, the UGN-301 dose will begin at least 1 dose level lower than the highest dose level cleared in the monotherapy arm, or 1 dose level lower than the RP2D. Eligible patients in each study treatment arm will enter a 12-week Induction Period. Patients with noninvasive papillary carcinoma and/or tumor that invades the lamina propria (Ta and/or T1) who do not have disease recurrence and patients with carcinoma in situ (CIS) who have a complete response (CR) at 3 months after the start of treatment will return to the clinic for a Safety Follow-up Visit at 6 months after the start of treatment. Ta/T1 patients without disease recurrence and CIS patients with CR at 6 months may enter an Optional Maintenance Period of up to 9 months.

Conditions

• Non-muscle Invasive Bladder Cancer
• NMIBC
• Carcinoma in Situ of Bladder
• Bladder Cancer
• Urothelial Carcinoma Bladder
• Urothelial Carcinoma Recurrent

Interventions

Type	Name	Description
DRUG	UGN-301	Induction Period: Intravesical administration once weekly for 6 weeks. Optional Maintenance Period: Intravesical administration once every 3 months (at 6, 9, and 12 months after the start of treatment).
DRUG	UGN-201	Induction Period: Intravesical administration once weekly for 6 weeks. Optional Maintenance Period: Intravesical administration once every 3 months (at 6, 9, and 12 months after the start of treatment).
DRUG	Gemcitabine	Induction Period: Intravesical administration once weekly for 6 weeks. Optional Maintenance Period: Intravesical administration once every 3 months (at 6, 9, and 12 months after the start of treatment).

Timeline

• Start date: 2022-06-01
• Primary completion: 2026-01-09
• Completion: 2026-01-09
• First posted: 2022-05-17
• Last updated: 2026-01-14

Locations

13 sites across 3 countries: United States, Italy, Spain

Regulatory

• FDA-regulated drug study