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UGN-104

Phase 3

Upper Urinary Tract Urothelial Carcinoma | Small molecule | Oncology |UroGen Pharma Ltd.|Last Updated: Jun 16, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment70

FDA Designations

No designations recorded

Clinical trial landscape

UGN-104 · 1 trial · 2 indications

Phase 3 1
NCT06774131A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial CancerUpper Urinary Tract Urothelial Carcinoma
RECRUITING70 Analytics
PHASE3RECRUITING
A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer
Upper Urinary Tract Urothelial CarcinomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Complete response rate (CRR)
3 months

CRR is defined as the proportion of patients who achieved CR at the PDE Visit.

Secondary Endpoints

Duration of response (DOR)
12 months
Durable complete response (DCR) rate
12 months
Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, TEAEs of special interest, and abnormal clinical laboratory test results (hematology and serum chemistry)
15 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
UGN-104EXPERIMENTALPatients 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.

Interventions

NameTypeDescription
UGN-104DRUGUGN-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.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites62

Inclusion Criteria: 1. Provide written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and protocol. 2. Naive or recurrent patients with LG, non-invasive UTUC in the pyelocalyceal system, with the following disease characteristi...

Countries:United StatesBulgariaGeorgiaHungaryIsraelLatviaPolandRomaniaSpain
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Recent Changes (Last 90 Days)

LOWJun 16, 2026NCT06774131lastUpdatePostDate: changed
LOWJun 16, 2026NCT06774131lastUpdatePostDate: changed
LOWJun 16, 2026NCT06774131lastUpdatePostDate: changed
MEDIUMMay 26, 2026NCT06774131primaryCompletionDate: changed
LOWMay 24, 2026NCT06774131studyFirstPostDate: changed

Frequently asked questions about UGN-104

What is UGN-104 used for?

UGN-104 is an investigational small molecule being developed for the treatment of low-grade Upper Tract Urothelial Cancer (UTUC), a type of urothelial carcinoma. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.

Who makes UGN-104?

UGN-104 is being developed by UroGen Pharma Ltd., a biopharmaceutical company traded on NASDAQ under the ticker URGN. The company is conducting a Phase 3 clinical trial to evaluate the drug's safety and efficacy in patients with low-grade upper tract urothelial cancer.

What phase is UGN-104 in?

UGN-104 is currently in Phase 3 clinical development. It is being studied in a single-arm, open-label trial that is actively recruiting participants. The drug is investigational and has not yet received FDA approval.

What clinical trials is UGN-104 in?

UGN-104 is being evaluated in a Phase 3 single-arm study (NCT06774131) for the treatment of low-grade Upper Tract Urothelial Cancer. The trial is recruiting 70 participants across multiple countries, including the United States, Bulgaria, Georgia, Hungary, Israel, Latvia, Poland, Romania, and Spain.

Is UGN-104 FDA approved?

UGN-104 is not FDA approved. It is an investigational drug currently in Phase 3 clinical trials. The ongoing study is designed to assess its safety and efficacy in patients with low-grade upper tract urothelial cancer, but regulatory approval has not been granted.