Treatment of low-grade non-muscle invasive bladder cancer with UGN-103

A Phase 3, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of UGN-103, a Novel Formulation of UGN-102, for the Treatment of Patients With Low-grade (LG) Non-muscle Invasive Bladder Cancer (NMIBC) at Intermediate-risk (IR) of Recurrence

This study is testing a new treatment called UGN-103 for people with low-grade non-muscle invasive bladder cancer to see if it can help them get better.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 92 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: UroGen Pharma Ltd. (industry)
• Drugs / interventions: chemotherapy, immunotherapy, radiation
• Locations: 14 sites (San Diego, California and 13 other locations)
• Trial ID: NCT06331299 on ClinicalTrials.gov

What this trial studies

This Phase 3, single-arm, multicenter study evaluates the efficacy and safety of UGN-103, a novel formulation, for patients with low-grade non-muscle invasive bladder cancer (LG-NMIBC). Participants will receive UGN-103 instilled in the bladder once weekly for six weeks, totaling six doses. The primary outcome is the complete response rate assessed at three months through visual observation, histopathology, and urine cytology. Patients with a complete response will enter a follow-up period for ongoing evaluation, while those with a non-complete response will receive standard care.

Who should consider this trial

Good fit: Ideal candidates are patients with histologically confirmed low-grade non-muscle invasive bladder cancer and a history of at least one prior episode of NMIBC.

Not a fit: Patients with high-grade disease or those who do not meet the specific eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for patients with low-grade non-muscle invasive bladder cancer.

How similar studies have performed: Other studies have shown promise with similar approaches, but this specific formulation and its application in this patient population is being evaluated for the first time.

Eligibility criteria

Inclusion Criteria:

1. Provide written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and the protocol.
2. Patient who has LG-NMIBC (Ta) histologically confirmed by cold cup biopsy at Screening or within 8 weeks before Screening.
3. History of at least 1 prior episode of NMIBC. Note: This refers to a previous episode(s) and not to the current episode for which the patient is being screened.
4. Has intermediate-risk disease, defined as having 1 or 2 of the following:

   * Presence of multiple tumors.
   * Solitary tumor \> 3 cm.
   * Early or frequent recurrence (≥ 1 occurrence of LG-NMIBC within 1 year of the current diagnosis at the initial Screening Visit).
5. Negative voiding cytology for high-grade (HG) disease within 8 weeks before Screening.
6. Has adequate organ and bone marrow function as determined by routine laboratory tests:

   * Leukocytes ≥ 3,000/μL.
   * Absolute neutrophil count ≥ 1,500/μL.
   * Platelets ≥ 100,000/μL.
   * Hemoglobin ≥ 9.0 g/dL.
   * Total bilirubin ≤ 1.5 × upper limit of normal (ULN).
   * Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN.
   * Alkaline phosphatase (ALP) ≤ 2.5 × ULN.
   * Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min.
7. Has an anticipated life expectancy of at least the duration of the trial.
8. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. Women of childbearing potential (defined as premenopausal women who have not been sterilized), including female patients and female partners of male patients, must be willing to use 2 acceptable forms of effective contraception from enrollment through 6 months post-treatment.

Exclusion Criteria:

1. Received Bacillus Calmette-Guérin (BCG) treatment for urothelial carcinoma (UC) within the previous 1 year.
2. History of HG bladder cancer (papillary or carcinoma in situ) in the past 2 years.
3. Known allergy or sensitivity to any component of the study treatment (including excipients) that in the investigator's opinion cannot be readily managed.
4. Clinically significant urethral stricture that would preclude passage of a urethral catheter.
5. History of:

   * Neurogenic bladder.
   * Active urinary retention.
   * Any other condition that would prohibit normal voiding.
6. Past or current muscle invasive bladder cancer (ie, T2, T3, T4) or metastatic UC.
7. Current tumor stage of T1.
8. Concurrent upper tract urothelial carcinoma (UTUC).
9. Evidence of active urinary tract infection (UTI) that in the investigator's opinion cannot be treated and resolved prior to biopsy and/or administration of study treatment.
10. Is pregnant or breastfeeding.
11. Has an underlying substance abuse or psychiatric disorder such that, in the opinion of the investigator, the patient would be unable to comply with the protocol.
12. History of prior treatment with an intravesical chemotherapeutic agent in the past 2 years except for a single dose of chemotherapy immediately after any previous transurethral resection of bladder tumors (TURBT).
13. Has participated in a study with an investigational agent or device within 30 days of enrollment.
14. Has previously participated in a study in which they received UGN-102.
15. Has any other active malignancy requiring treatment with systemic anticancer therapy (eg, chemotherapy, immunotherapy, radiation therapy). Superficial cancers such as cutaneous basal cell or squamous cell carcinomas that can be treated locally are allowed.
16. Has any other clinically significant medical or surgical condition that in the investigator's opinion could compromise patient safety or the interpretation of study results.
17. Where applicable per country regulation, the patient must not be currently committed to an institution by virtue of an order issued by either judicial or administrative authorities.

Where this trial is running

San Diego, California and 13 other locations

• Genesis Research, LLC — San Diego, California, United States (RECRUITING)
• Peachtree Clinical Solutions — Powder Springs, Georgia, United States (RECRUITING)
• Indiana University Melvin and Bren Simon Cancer Center — Indianapolis, Indiana, United States (RECRUITING)
• Garden State Urology — Morristown, New Jersey, United States (RECRUITING)
• Great Lakes Physician dba WNYU — Cheektowaga, New York, United States (RECRUITING)
• AccuMed Research Associates — Garden City, New York, United States (RECRUITING)
• Crystal Run Healthcare — Middletown, New York, United States (RECRUITING)
• Laura and Isaac Perlmutter Cancer Center at NYU Langone — New York, New York, United States (RECRUITING)
• Perlmutter Cancer Center at NYU Langone Hospital - Long Island — New York, New York, United States (RECRUITING)
• Carolina Urologic Research Center — Myrtle Beach, South Carolina, United States (RECRUITING)
• Urology Associates P.C. - Nashville — Nashville, Tennessee, United States (RECRUITING)
• Houston Metro Urology (HMU) - Southwest Location — Houston, Texas, United States (RECRUITING)
• Houston Methodist Hospital — Houston, Texas, United States (RECRUITING)
• Spokane Urology, P.S. — Spokane, Washington, United States (RECRUITING)

Study contacts

• Study coordinator: Karrie Hilsinger

Conditions: Bladder Cancer, Urothelial Carcinoma, Urothelial Carcinoma Bladder, Non-muscle invasive bladder cancer, Low-grade non-muscle invasive bladder cancer, Intermediate-risk non-muscle invasive bladder cancer, NMIBC, UGN-103