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Study of UGN-104 (mitomycin) treatment for patients with low-grade upper tract urothelial cancer

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial focuses on patients with Low-grade Upper Tract Urothelial Cancer, a type of cancer that develops in the lining of the kidney's collecting system. The study will test a new medicine called UGN-104, which contains the active substance mitomycin. This treatment comes in the form of a powder that is mixed into a solution before use.

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 directly into the upper urinary tract through a procedure called intravesical administration. Each patient will receive six treatments of the medicine over a treatment period that can last up to 12 months.

After completing the treatment, patients will have a check-up approximately 5 weeks after their final dose to determine if the treatment was successful in eliminating the cancer. The study will continue to monitor patients to see how long the treatment effects last and to check for any side effects. Throughout the study, doctors will perform various tests to ensure patient safety, including blood tests and imaging of the urinary tract.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Your medical team will perform a detailed evaluation to confirm low-grade upper tract urothelial cancer diagnosis

    Laboratory tests will be conducted to check organ function and blood counts

    A visual examination of tumors in the upper urinary tract will be performed

  2. Step 2

    Treatment preparation

    The medical team will ensure there is no active urinary tract infection before starting treatment

    If infection is present, antibiotics will be prescribed and treatment will be delayed until resolved

  3. Step 3

    Treatment administration

    UGN-104 medication will be administered directly into the upper urinary tract

    You will receive six treatments in total

    The medication is a special powder form that is prepared for administration

  4. Step 4

    Post-treatment evaluation

    5 weeks (plus/minus 1 week) after the sixth treatment, a detailed examination will be performed

    This examination includes: urine tests, visual examination of the urinary tract, and if needed, a tissue sample

    The medical team will assess if the treatment has been effective in removing the cancer

  5. Step 5

    Follow-up period

    Regular check-ups will be scheduled to monitor your condition

    Safety assessments will include blood tests, urine analysis, physical examinations, and vital signs monitoring

    You will be asked to complete questionnaires about your health and treatment experience

  6. Step 6

    Safety considerations

    Male patients must use appropriate contraception for 48 hours after each treatment

    All patients must use two forms of effective contraception during the study and for 6 months afterward

    Regular monitoring of side effects and any health changes will continue throughout the study

Who can join the trial?

17 criteria

  • Must provide written informed consent and agree to follow study requirements
  • Must be 18 years or older
  • Must have low-grade, non-invasive upper tract urothelial cancer in the kidney collection system with:
    • At least 1 measurable tumor no larger than 15 millimeters
    • At least 1 tumor must be at least 5 millimeters in size after biopsy
    • Biopsy confirming low-grade cancer
    • Urine test showing no signs of high-grade cancer
    • Must have good physical function status (ECOG score less than 3, which means able to care for self but unable to work)
    • Must have life expectancy of more than 24 months
    • Must have adequate organ function shown by blood tests including:
      • Normal white blood cell counts
      • Normal platelet levels
      • Adequate hemoglobin levels
      • Normal liver function tests
      • Adequate kidney function
      • Must not have active urinary tract infection
      • Must agree to use effective birth control:
        • Both men and women must use two forms of birth control during study
        • Men must use condoms for 48 hours after each treatment
        • Birth control must continue for 6 months after treatment ends

Who cannot join the trial?

12 criteria

  • Age under 18 years old
  • Patients with high-grade urothelial cancer (more aggressive form of cancer in the urinary tract)
  • Previous treatment with chemotherapy or immunotherapy within the last 6 months
  • Presence of metastatic disease (cancer that has spread to other parts of the body)
  • Severe kidney dysfunction
  • Active urinary tract infection
  • Pregnant or breastfeeding women
  • History of allergic reactions to similar medications
  • Participation in another clinical trial within the last 30 days
  • Significant heart, liver, or other medical conditions that could interfere with study participation
  • Unable to follow study procedures or attend required visits
  • Current substance abuse or mental health conditions that could affect compliance
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Investigated drugs

UGN-104 is an investigational medication delivered directly to the upper urinary tract. It is a novel formulation of UGN-101, designed to treat low-grade upper tract urothelial cancer. This medication is applied locally to target cancer cells in the urinary system, specifically in the upper parts of the urinary tract. The medication is being studied as a potential treatment option that may help eliminate tumors in this specific area of the body.

What is already known about the treatment

  • UGN-104

    A novel formulation of UGN-101, administered locally in the upper urinary tract for the treatment of Low-grade Upper Tract Urothelial Cancer (LG-UTUC). This investigational medication is currently being studied in Phase 3 clinical trials as a targeted therapy for urothelial tumors. The drug is designed to deliver medication directly to the affected area of the urinary tract, providing localized treatment while minimizing systemic exposure. As a novel formulation, it represents an advancement in the development of targeted therapies for urological cancers, with the primary goal of achieving tumor ablation in patients with LG-UTUC.

  • UGN-101

    The original formulation that serves as the basis for UGN-104, developed for the treatment of low-grade upper tract urothelial cancer. This medication is administered through direct instillation into the upper urinary tract, allowing for targeted treatment of cancerous tissues. It belongs to the class of locally-administered anti-cancer medications and is specifically designed to treat tumors in the upper urinary tract system, providing a non-surgical treatment option for patients with LG-UTUC.

Investigated diseases

Low-grade Upper Tract Urothelial Cancer - A rare form of cancer that develops in the lining of the upper urinary tract, specifically in the renal pelvis or ureter. This cancer begins in the urothelial cells (also called transitional cells) that line these structures. The term "low-grade" indicates that the cancer cells appear more similar to normal cells and tend to grow more slowly than high-grade cancers. The disease typically develops gradually, with cancer cells growing within the inner lining of the urinary tract. It can occur in one or multiple locations within the upper urinary system.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-519343-15-00Protocol codeUT002Estimated enrolment104 patientsSponsorUrogen Pharma Ltd.

sourced from the EU Clinical Trials Register and site verification

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