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Phase 1 Study of UGN-501 for Recurrent Non-Muscle Invasive Bladder Cancer

This study is testing a new treatment called UGN-501 for adults with recurrent non-muscle invasive bladder cancer (NMIBC). UGN-501 is given directly into the bladder. The main goals are to find out how safe UGN-501 is, what side effects it might cause, and to determine the best dose for future studies. Researchers will also look at how much UGN-501 is in your blood and urine, and if it helps to clear the cancer. You might be able to join if you are 18 or older and have confirmed recurrent NMIBC, including high-grade or low-grade disease, or carcinoma in situ (CIS). The study is currently unclear on its recruitment status.

Study design
This is a Phase 1, open-label study, meaning you and your doctors will know you are receiving UGN-501. It is a dose-escalation study, planning to enroll about 30 participants.
What's involved
You would receive UGN-501 instilled into your bladder weekly for 6 weeks. If eligible, you might then receive 3 weekly instillations every three months for up to a year. You will have disease assessments every 3 months for up to 15 months.
Compensation
Not stated in the trial record.
Follow-up
You will be followed for safety and side effects for up to 15 months. Researchers will also measure UGN-501 levels in your blood and urine for up to 12 weeks.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07725796

A Phase 1 Dose-escalation Study of UGN-501 Administered Intravesically in Adult Participants With Recurrent NMIBC

Not Yet Recruiting
PHASE1Ages 18+InterventionalTreatment
UroGen Pharma, Inc., a subsidiary of UroGen Pharma Ltd.
~30 participants
Updated 2026-07-24 on ClinicalTrials.gov
What's tested:UGN-501

At a glance

Recruiting sites
0 of 11 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs)
Measured over Up to 15 Months
+3 more outcomes measured

Conditions

Bladder (Urothelial, Transitional Cell) Cancer
NMIBC
Urothelial Carcinoma Bladder
Urothelial Carcinoma in Situ
Urothelial Carcinoma Recurrent
Urothelial Carcinoma of the Urinary Bladder
Non-Muscle Invasive Bladder Carcinoma
Non-muscle Invasive Bladder Cancer (NMIBC)

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NCT07725796

Where you'd take part

This study runs at 11 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Brigham and Women's Hospital

    Boston, Massachusettsstudy coordinator listed

  • East Valley Urology Center of Arizona

    Queen Creek, Arizonastudy coordinator listed

  • Ichan School of Medicine at Mount Sinai

    New York, New Yorkstudy coordinator listed

  • Michael G Oefelein Clinical Trials

    Bakersfield, Californiastudy coordinator listed

  • START Carolinas

    Myrtle Beach, South Carolinastudy coordinator listed

  • SUNY Upstate Medical University

    Syracuse, New Yorkstudy coordinator listed

  • University of Nebraska Medical Center

    Omaha, Nebraskastudy coordinator listed

  • University of Pennsylvania-Perelman Center for Advanced Medicine

    Philadelphia, Pennsylvaniastudy coordinator listed

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Sebastian Mirkin, MD · STUDY_DIRECTOR · UroGen Pharma

Who to contact

Heather Lansford
[email protected]
610-226-5111
Email the study team

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Eligibility criteria

Inclusion

Has BCG-unresponsive disease, defined as:
persistent or recurrent CIS alone or with recurrent Ta/T1 disease within 12 months of completion of adequate BCG therapy; or
recurrent high-grade Ta/T1 disease within 6 months of completion of adequate BCG therapy; or
high-grade T1 disease at the first evaluation following a BCG induction course.
Is BCG-exposed, defined as high-grade persistent or recurrent NMIBC within 24 months of the last dose of BCG but not meeting the definition of BCG-unresponsive disease, and received at least 5 of 6 doses of an initial induction course of BCG.
Is BCG intolerant, defined as the inability to tolerate at least 1 full induction course of BCG.
Has a high-grade Ta tumor ≤3 cm and failed at least 1 previous course of therapy, such as TURBT plus an adjuvant induction course of intravesical chemotherapy. 4. Participants with low-grade disease must meet one of the following criteria:
Ta tumors recurring within 1 year.
Ta solitary tumor \>3 cm.
Ta multifocal tumors.
T1 tumors. 5. All visible papillary tumors must be resected and obvious areas of CIS fulgurated during Screening or within 6 weeks before Screening. Participants with T1 disease should have a re-staging TURBT during Screening or within 6 weeks before Screening. Enhanced cystoscopy, such as blue light cystoscopy or other locally accepted modalities, is permitted, but the same modality must be used for all subsequent disease assessments. 6. Has Eastern Cooperative Oncology Group performance status score ≤2. 7. Has no concomitant upper tract urothelial carcinoma (UTUC) or urothelial carcinoma within the prostatic stroma. Freedom from upper tract disease, if clinically indicated, must be demonstrated by no evidence of upper tract tumor by either IV pyelogram, retrograde pyelogram, CT urogram with or without contrast, MRI urogram with or without contrast, or ureteroscopy with ureteral washing for cytology, performed within 6 months of enrollment. Participants with urothelial carcinoma involving the prostatic urethra, where carcinoma is confined to the ducts and/or epithelium, may be included after a restaging TURBT is performed to rule out more extensive disease involving the prostatic stroma. 8. Participants with prostate cancer may be eligible if, after surgery or radiation, they do not meet criteria for biochemical recurrence, or if they are on active surveillance at very low, low, or favorable risk for progression, defined as Gleason Grade Group 1 or 2, Gleason score ≤7, prostate-specific antigen \<20 ng/dL, and cT1-cT2b, at the discretion of the investigator. 9. Has adequate organ and bone marrow function within 14 days of treatment initiation, as determined by routine laboratory tests:
Leukocytes ≥3000/μL.
Absolute neutrophil count ≥1500/μL.
Platelets ≥100,000/μL.
Hemoglobin ≥9.0 g/dL.
Total bilirubin ≤1.5 × upper limit of normal (ULN).
Aspartate aminotransferase (AST) ≤2.5 × UL
Alanine aminotrasferase (ALT) ≤2.5 × ULN.
Alkaline phosphatase ≤2.5 × ULN.
Estimated creatinine clearance ≥30 mL/min using the Cockcroft-Gault equation. 10. Has a life expectancy \>12 months. 11. Participants and participant partners must agree to follow contraception and barrier method requirements consistent with local regulations and the protocol during the study intervention period and for at least 12 weeks after the last study intervention instillation. 12. Has signed informed consent and is willing and able to comply with the requirements and restrictions listed in the informed consent form and protocol.

What this trial measures

  • Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs)Up to 15 Months

    The number of participants with each type of event will be summarized.

  • Concentration of UGN-501 in blood and urineUp to 12 Weeks

    Data will be summarized using descriptive statistics.

  • Complete response rate (CRR)3 Months

    CRR is defined as the proportion of CIS participants who achieved CR at the Week 12 (3-month) Visit.

  • Recurrence-free survival (RFS) rate6 Months

    RFS rate is defined as the proportion of participants with Ta/T1 disease who are recurrence-free at the 6-month Visit.