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

NCT07725796 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: UGN-501-101
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 11

Brief Summary

This study is being conducted to evaluate the safety and tolerability of UGN-501 administered intravesically in adult participants with recurrent non-muscle invasive bladder cancer (NMIBC) and to determine the recommended Phase 2 dose (RP2D).

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)

Keywords

CIS; Ta bladder cancer; T1 bladder cancer; BCG-unresponsive NMIBC; BCG-exposed NMIBC; BCG-intolerant NMIBC; Intravesical therapy; Oncolytic Virus

Outcome Measures

Primary Outcomes (4)

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

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

  Up to 15 Months

• Concentration of UGN-501 in blood and urine

  Data will be summarized using descriptive statistics.

  Up to 12 Weeks

• Complete response rate (CRR)

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

  3 Months

• Recurrence-free survival (RFS) rate

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

  6 Months

Secondary Outcomes (7)

• Presence of anti-drug antibodies (ADA) in serum

  Up to 12 Weeks

• UGN-501 maximum concentration (Cmax) following single and repeat dose administration

  Up to 12 Weeks

• UGN-501 area under the concentration-time curve (AUC) following single and repeat dose administration

  Up to 12 Weeks

• UGN-501 terminal half-life (t1/2) following single and repeat dose administration

  Up to 12 Weeks

• UGN-501 time to maximum concentration (tmax) following single and repeat dose administration

  Up to 12 Weeks

Study Arms (1)

UGN-501 Dose Escalation (Part 1)

EXPERIMENTAL

Dose escalation of UGN-501 in participants with recurrent NMIBC with high grade (HG) Ta and/or T1 disease and/or CIS or recurrent low grade (LG) Ta and/or T1 disease.

Biological: UGN-501

Interventions

UGN-501 BIOLOGICAL

UGN-501 is administered by intravesical instillation into the bladder. Participants receive 6 once-weekly instillations during the Induction Period. Participants eligible for maintenance receive 3 once-weekly instillations quarterly from Month 3 through Month 12.

UGN-501 Dose Escalation (Part 1)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must be 18 years of age or older at the time of signing informed consent.
• Has confirmed recurrent non-muscle invasive bladder cancer (NMIBC) with high-grade Ta and/or T1 disease and/or carcinoma in situ (CIS), or recurrent low-grade Ta and/or T1 disease.
• Participants with high-grade Ta and/or T1 disease and/or CIS must meet one of the following criteria:
• 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.
• Adequate BCG therapy is defined as at least 5 of 6 doses of an initial induction course plus either at least 2 of 3 doses of maintenance therapy or at least 2 of 6 doses of a second induction course. Participants with BCG-unresponsive disease also must be unwilling or unfit to undergo radical cystectomy.
• 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.
• 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.

You may not qualify if:

• Current or previous evidence of muscle invasive, locally advanced nonresectable, or metastatic urothelial carcinoma, including T2, T3, T4, and/or stage IV disease.
• Current systemic therapy for bladder cancer.
• Prior treatment with any human adenovirus-based therapy, such as nadofaragene firadenovec-vncg or cretostimogene grenadenorepvec.
• Intravesical therapy within 4 weeks before starting study intervention, including but not limited to BCG, chemotherapy, and nogapendekin alfa inbakicept.
• Participation in a study of an investigational agent with receipt of study therapy, or receipt of an investigational device, within 4 weeks before the first dose of study intervention.
• Receipt of immune modulator therapy within 5 half-lives of starting study intervention, including but not limited to pembrolizumab, BCG, and nogapendekin alfa inbakicept.
• Receipt of a vaccine or anti-viral agent within 2 weeks before starting study intervention.
• Active infection requiring systemic therapy, including urinary tract infection. Participants may enter the study once the infection is satisfactorily treated.
• Immunocompromised state, including but not limited to HIV infection or any condition that required systemic immunosuppressive treatment in the past 2 years, such as active systemic autoimmune disease or solid organ or stem cell transplant. Short courses (≤14 days) of steroids for medical reasons without anticancer intent, such as atopic dermatitis, psoriasis, infection, or allergic reaction, are permitted if the last dose was at least 4 weeks before the first dose of study intervention.
• Any medical, psychological, familial, sociological, or geographical condition that, in the opinion of the investigator, would preclude participation in the study.
• History of malignancy of another organ system within the past 5 years, except previously treated UTUC, basal cell carcinoma or squamous cell carcinoma of the skin, and/or prostate cancer meeting protocol-specified criteria.
• Cannot tolerate intravesical dosing or intravesical surgical manipulation.
• Known allergy or hypersensitivity to any of the study interventions or any study intervention excipients.

Sponsors & Collaborators

• UroGen Pharma, Inc., a subsidiary of UroGen Pharma Ltd.: lead

Study Sites (11)

East Valley Urology Center of Arizona

Queen Creek, Arizona, 85140, United States

Location

Michael G Oefelein Clinical Trials

Bakersfield, California, 93301, United States

Location

Urology Center of Southern California

Murrieta, California, 92563, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Ichan School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

University of Pennsylvania-Perelman Center for Advanced Medicine

Philadelphia, Pennsylvania, 19104, United States

Location

START Carolinas

Myrtle Beach, South Carolina, 29572, United States

Location

UPNT Research Institute

Arlington, Texas, 76017, United States

Location

Urology Austin

Austin, Texas, 78759, United States

Location

MeSH Terms

Conditions

Neoplasms; Non-Muscle Invasive Bladder Neoplasms

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Urinary Bladder Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases

Study Officials

• Sebastian Mirkin, MD

  UroGen Pharma

  STUDY DIRECTOR

Central Study Contacts

Heather Lansford

CONTACT

610-226-5111; heather.lansford@urogen.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 21, 2026
• First Posted: July 24, 2026
• Study Start (Estimated): September 30, 2026
• Primary Completion (Estimated): August 2, 2029
• Study Completion (Estimated): August 2, 2029
• Last Updated: July 24, 2026

Record last verified: 2026-07

Locations

US (11)