A Phase 3 Study of UGN-103 for Treatment of Patients With Low-grade Intermediate-risk Non-muscle Invasive Bladder Cancer

NCT ID: NCT06331299

Last Updated: 2026-01-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

99 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-08-29

Study Completion Date

2026-09-30

Brief Summary

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This Phase 3, single-arm, multicenter study will evaluate the efficacy and safety of UGN-103, a novel formulation of UGN-102, instilled in the urinary bladder of patients with low-grade non-muscle invasive bladder cancer (LG-NMIBC).

Detailed Description

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Eligible patients will be treated with UGN-103 once weekly for 6 weeks (a total of 6 doses).

Efficacy will be assessed by the complete response rate (CRR) at the 3-month Visit (approximately 3 months after the first instillation). Response will be determined based on visual observation (white light cystoscopy), histopathology of any remaining or new lesions by central pathology lab (if applicable), and interpretation of urine cytology by central pathology lab.

Patients who have a complete response (CR) at the 3-month Visit, defined as having no detectable disease (NDD) in the bladder, will enter the Follow-up Period of the study. During the Follow-up Period, patients will return to the clinic every 3 months for evaluation of response. Patients will remain on study until disease recurrence, disease progression, death, or the last patient completes 12 months of follow-up (ie, 12 months after the 3-month Visit), whichever occurs first.

Patients who have a non-complete response (NCR) at the 3-month Visit will undergo investigator designated standard of care (SOC) and have a separate End of Study (EOS) Visit performed.

Conditions

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Bladder Cancer Urothelial Carcinoma Urothelial Carcinoma Bladder

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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UGN-103

Patients will receive UGN-103 (75 mg mitomycin) once weekly for 6 weeks (a total of 6 doses).

Group Type EXPERIMENTAL

UGN-103

Intervention Type DRUG

UGN-103 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-103 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 bladder.

Interventions

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UGN-103

UGN-103 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-103 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 bladder.

Intervention Type DRUG

Other Intervention Names

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UGN-103 (mitomycin) for intravesical solution

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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UroGen Pharma Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sebastian Mirkin, MD

Role: STUDY_DIRECTOR

UroGen Pharma

Locations

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Loma Linda University Medical Center

Loma Linda, California, United States

Site Status

Genesis Research, LLC

San Diego, California, United States

Site Status

The George Washington University Medical Faculty Associates

Washington D.C., District of Columbia, United States

Site Status

Peachtree Clinical Solutions

Powder Springs, Georgia, United States

Site Status

Indiana University Melvin and Bren Simon Cancer Center

Indianapolis, Indiana, United States

Site Status

Minnesota Urology - Metro Urology - Woodbury

Woodbury, Minnesota, United States

Site Status

Garden State Urology

Morristown, New Jersey, United States

Site Status

Great Lakes Physician dba WNYU

Cheektowaga, New York, United States

Site Status

AccuMed Research Associates

Garden City, New York, United States

Site Status

Crystal Run Healthcare

Middletown, New York, United States

Site Status

Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, United States

Site Status

Perlmutter Cancer Center at NYU Langone Hospital - Long Island

New York, New York, United States

Site Status

Carolina Urologic Research Center

Myrtle Beach, South Carolina, United States

Site Status

Urology Associates P.C. - Nashville

Nashville, Tennessee, United States

Site Status

Houston Metro Urology (HMU) - Southwest Location

Houston, Texas, United States

Site Status

Houston Methodist Hospital

Houston, Texas, United States

Site Status

Spokane Urology, P.S.

Spokane, Washington, United States

Site Status

Multiprofile Hospital for Active Treatment "Dr. Tota Venkova", Gabrovo, Department of Urology

Gabrovo, Gabrovo, Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment "Dr. Georgi Stranski", Pleven, Urology Clinic

Pleven, Pleven Province, Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv, Clinic of Urology

Plovdiv, Plovdiv Province, Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment, Plovdiv, Clinic of Urology

Plovdiv, Plovdiv Province, Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment 'Kanev', Ruse, Department of Urology

Rousse, Ruse Province, Bulgaria

Site Status

Multiprofile Hospital for Active Treatment - Shumen, Department of Urology

Shumen, Shumen Province, Bulgaria

Site Status

University Multiprofile Hospital for Active Treatment and Emergency Medicine "N.I. Pirogov", Sofia, Urology Clinic

Sofia, Sofia-Grad, Bulgaria

Site Status

West Tallinn Central Hospital Ltd., Department of Urology

Tallinn, Harju, Estonia

Site Status

North Estonia Medical Centre Foundation Ltd.

Tallinn, Harju, Estonia

Site Status

East Viru Central Hospital, Surgery Clinic

Kohtla-Järve, Ida-Virumaa, Estonia

Site Status

Tartu University Hospital, Surgery Clinic, Department of Urology and Kidney Transplantation

Tartu, Tartu County, Estonia

Site Status

LLC "Todua Clinic"

Tbilisi, , Georgia

Site Status

Geo Hospitals LLC

Tbilisi, , Georgia

Site Status

Tbilisi State Medical University and Ingorokva High Medical Technology University Clinic LLC

Tbilisi, , Georgia

Site Status

LTD MMT Hospital

Tbilisi, , Georgia

Site Status

JSC Jerarsi

Tbilisi, , Georgia

Site Status

Daugavpils Regional Hospital, Urology Department

Daugavpils, , Latvia

Site Status

Liepajas Regional Hospital, Urology Department

Liepāja, , Latvia

Site Status

P. Stradins Clinical University Hospital, Center for Urology

Riga, , Latvia

Site Status

LLC Riga East University Hospital, Clinic of Urology and Oncologic Urology

Riga, , Latvia

Site Status

Bihor County Emergency Clinical Hospital

Oradea, Bihor County, Romania

Site Status

Brasov County Emergency Clinical Hospital, Department of Urology

Brasov, Brașov County, Romania

Site Status

Craiova County Emergency Clinical Hospital, Department of Urology

Craiova, Dolj, Romania

Site Status

SC Clinica Polisano Hospital

Sibiu, Sibiu County, Romania

Site Status

Colentina Clinical Hospital, Department of Urology

Bucharest, , Romania

Site Status

"Prof. Dr. Th. Burghele" Clinical Hospital, Department of Urology III

Bucharest, , Romania

Site Status

University Hospital Virgen de la Victoria

Málaga, Andalusia, Spain

Site Status

Puigvert Foundation

Barcelona, Catalonia, Spain

Site Status

University Hospital Foundation Jimenez Diaz

Madrid, Madrid, Spain

Site Status

University Hospital 12 de Octubre

Madrid, Madrid, Spain

Site Status

La Paz University Hospital

Madrid, Madrid, Spain

Site Status

Countries

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United States Bulgaria Estonia Georgia Latvia Romania Spain

Other Identifiers

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BL013

Identifier Type: -

Identifier Source: org_study_id

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