UGN-104 for Transitional Cell Carcinoma

Recruiting at 2 trial locations

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests UGN-104, a new treatment for individuals with low-grade upper tract urothelial cancer, a type of bladder cancer. The trial aims to evaluate the effectiveness and safety of UGN-104. Participants with a small, visually measurable tumor in their upper urinary tract, who have not undergone certain past treatments or had specific types of cancer, may qualify for this study. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants the opportunity to contribute to a treatment that could soon become widely available.

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.

Is there any evidence suggesting that UGN-104 is likely to be safe for humans?

Research shows that UGN-104 is a new version of UGN-101, already approved in the United States and Israel as JELMYTO for treating a different type of cancer. This approval indicates that its main ingredient, mitomycin, is considered safe for certain uses in people.

Although UGN-104 is still under study, the approval of a similar treatment suggests it might be safe. However, since UGN-104 is being tested for a new purpose, its safety profile might differ. The study is in an advanced stage, which usually indicates earlier tests found it to be well-tolerated, but specific safety details for UGN-104 are not provided.

Participants should consider these points and consult their healthcare provider if they have any concerns. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for transitional cell carcinoma, which often include surgery, chemotherapy, or immunotherapy, UGN-104 is delivered directly to the urinary tract. This unique delivery method uses a sterile hydrogel to administer mitomycin, allowing for targeted treatment directly to the cancer site. Researchers are excited about UGN-104 because this localized approach may reduce systemic side effects and improve drug effectiveness by concentrating the treatment exactly where it's needed. Additionally, the option for maintenance dosing could help sustain remission, offering a new potential strategy in managing this type of cancer.

What evidence suggests that UGN-104 might be an effective treatment for transitional cell carcinoma?

Research has shown that UGN-104 is a new version of UGN-101, which is already approved for treating low-grade upper tract urothelial cancer, a type of bladder cancer. In studies, UGN-101 demonstrated promising results: 36% of patients showed no signs of cancer after treatment, and 23% experienced a reduction in their cancer. This trial will evaluate UGN-104 for its potential effectiveness in treating transitional cell carcinoma, building on the success of UGN-101. ^{1 2 3 6 7}

Who Is on the Research Team?

Sebastian Mirkin, MD

Principal Investigator

UroGen Pharma

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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). 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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06774131

A Phase 3 Single-arm Study of UGN-104 for the Treatment ... 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] ...

2. investors.urogen.com investors.urogen.com/static-files/fba40caa-c888-42fa-9f09-545ac8346efd

Transforming Urothelial Cancer Care UGN-103, UGN-104, and UGN-501 are investigational drugs in development. The safety and efficacy of these agents have not been established by any regulatory body ...

3. withpower.com withpower.com/trial/ugn-104-for-transitional-cell-carcinoma-cb29b

UGN-104 for Transitional Cell Carcinoma 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 \[mitomycin\] ...

4. urotoday.com urotoday.com/recent-abstracts/urologic-oncology/bladder-cancer/164474-urogen-reports-third-quarter-2025-financial-results-as-zusduri-tm-launch-gains-momentum.html

UroGen Reports Third Quarter 2025 Financial Results as ... Reported three-month complete response rate of 77.8% from the Phase 3 UTOPIA trial of UGN-103, consistent with ENVISION results; ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7265178/

Management of Low-grade Upper Tract Urothelial Carcinoma Complete response, partial response, and no response were reported in 36%, 23%, and 9% of patients, respectively. Among the patients with complete response, 63% ...

6. sec.gov sec.gov/Archives/edgar/data/1668243/000119312524136728/d804276dex991.htm

EX-99.1 UroGen plans to initiate Phase 3 studies to explore the safety and efficacy of UGN-103 in LG-IR-NMIBC in 2024 and UGN-104 in LG-UTUC shortly thereafter.

7. clinicaltrials.eu clinicaltrials.eu/trial/study-of-ugn-104-mitomycin-treatment-for-patients-with-low-grade-upper-tract-urothelial-cancer/

Study of UGN-104 (mitomycin) treatment for patients with ... The purpose of this research is to evaluate how effective UGN-104 is at removing tumors in patients with this type of cancer. The medication will be given ...