UGN-104 for Transitional Cell Carcinoma

Overseen By Yafit Makmal

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: UroGen Pharma Ltd.

Must not be taking: Systemic chemotherapy

Disqualifiers: Invasive UC, Carcinoma in situ, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 10

Duration: 6 weeks

50 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, you cannot be treated with systemic chemotherapy during the study.

What is the purpose of this trial?

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).

Research Team

Sunil Raju, MBBS

Principal Investigator

UroGen Pharma

Eligibility Criteria

This trial is for patients with low-grade upper tract urothelial cancer, which affects the urinary system. Participants should have a diagnosis of this specific type of cancer and be suitable for treatment with UGN-104 instilled directly into the upper urinary tract.

Inclusion Criteria

My cancer is in the early stages and located in the upper urinary tract.

Provide written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and protocol

Patients with life expectancy > 24 months at time of Screening

Exclusion Criteria

I am currently undergoing or plan to undergo chemotherapy during the study.

I cannot receive treatment due to a blockage in my urinary system.

I am not pregnant, planning to become pregnant, or breastfeeding.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Eligible patients will be treated with UGN-104 once weekly for 6 weeks

6 weeks

6 visits (in-person)

Primary Disease Evaluation (PDE) Visit

Efficacy assessed by complete response rate (CRR) approximately 3 months after the first instillation

3 months

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with optional maintenance treatment

12 months

Every 3 months (in-person)

Treatment Details

Interventions

• UGN-104

Trial Overview The study is testing UGN-104, a new formulation related to JELMYTO (mitomycin), designed to treat low-grade upper tract urothelial cancer by being placed directly in the affected area within the urinary system.

Participant Groups

Group I: UGN-104 Experimental Treatment 1 Intervention

Patients will receive UGN-104 once weekly for 6 weeks (a total of 6 doses). The dose of UGN-104 to be instilled is 4 mg mitomycin per 1 mL sterile hydrogel via ureteral catheter (retrograde administration) or nephrostomy tube (antegrade administration), with total instillation volume based on volumetric measurements using pyelography, not to exceed 15 mL (60 mg mitomycin). Patients who have a CR at the PDE Visit will enter the Follow-up Period and may receive UGN-104 as maintenance treatment once monthly for 11 months (a total of 11 doses) at the discretion of the investigator.

Who Is Running the Clinical Trial?

UroGen Pharma Ltd.

Lead Sponsor

Trials

20

Recruited

1,400+