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

NCT06331299 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: BL013
• Study Type: interventional
• Target: 99
• Locations: 6 countries
• Sites: 44

Brief Summary

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).

Conditions

Bladder Cancer; Urothelial Carcinoma; Urothelial Carcinoma Bladder

Keywords

Non-muscle invasive bladder cancer; Low-grade non-muscle invasive bladder cancer; Intermediate-risk non-muscle invasive bladder cancer; NMIBC; UGN-103; UGN-102; Mitomycin

Outcome Measures

Primary Outcomes (1)

• Complete response rate (CRR)

  CRR is defined as the proportion of patients who achieved CR at the 3-month Visit.

  3 months

Secondary Outcomes (5)

• Duration of response (DOR)

  Up to 21 months

• Durable complete response (DCR) rate

  Up to 21 months

• Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, TEAEs of special interest, and abnormal clinical laboratory test results (hematology and serum chemistry)

  Up to 21 months

• Mitomycin plasma concentrations

  0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-103.

• Mitomycin maximum plasma concentration (Cmax)

  0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-103.

Study Arms (1)

UGN-103

EXPERIMENTAL

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

Drug: UGN-103

Interventions

UGN-103 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.

Also known as: UGN-103 (mitomycin) for intravesical solution

UGN-103

Eligibility Criteria

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

You may qualify if:

• Provide written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and the protocol.
• Patient who has LG-NMIBC (Ta) histologically confirmed by cold cup biopsy at Screening or within 8 weeks before Screening.
• 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.
• 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).
• Negative voiding cytology for high-grade (HG) disease within 8 weeks before Screening.
• 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.

You may not qualify if:

• Received Bacillus Calmette-Guérin (BCG) treatment for urothelial carcinoma (UC) within the previous 1 year.
• History of HG bladder cancer (papillary or carcinoma in situ) in the past 2 years.
• Known allergy or sensitivity to any component of the study treatment (including excipients) that in the investigator's opinion cannot be readily managed.
• Clinically significant urethral stricture that would preclude passage of a urethral catheter.
• History of:
• Neurogenic bladder.
• Active urinary retention.
• Any other condition that would prohibit normal voiding.
• Past or current muscle invasive bladder cancer (ie, T2, T3, T4) or metastatic UC.
• Current tumor stage of T1.
• Concurrent upper tract urothelial carcinoma (UTUC).
• 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.
• Is pregnant or breastfeeding.
• 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.
• 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).

Sponsors & Collaborators

• UroGen Pharma Ltd.: lead

Study Sites (48)

Loma Linda University Medical Center

Loma Linda, California, 92354, United States

Location

Genesis Research, LLC

San Diego, California, 92123, United States

Location

The George Washington University Medical Faculty Associates

Washington D.C., District of Columbia, 20037, United States

Location

Peachtree Clinical Solutions

Powder Springs, Georgia, 30127, United States

Location

Indiana University Melvin and Bren Simon Cancer Center

Indianapolis, Indiana, 46202, United States

Location

Minnesota Urology - Metro Urology - Woodbury

Woodbury, Minnesota, 55125, United States

Location

Garden State Urology

Morristown, New Jersey, 07962, United States

Location

Great Lakes Physician dba WNYU

Cheektowaga, New York, 14225, United States

Location

AccuMed Research Associates

Garden City, New York, 11530, United States

Location

Crystal Run Healthcare

Middletown, New York, 10941, United States

Location

Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States

Location

Perlmutter Cancer Center at NYU Langone Hospital - Long Island

New York, New York, 11501, United States

Location

Carolina Urologic Research Center

Myrtle Beach, South Carolina, 29572, United States

Location

Urology Associates P.C. - Nashville

Nashville, Tennessee, 37209-4035, United States

Location

Houston Metro Urology (HMU) - Southwest Location

Houston, Texas, 77027, United States

Location

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

Spokane Urology, P.S.

Spokane, Washington, 99202, United States

Location

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

Gabrovo, Gabrovo, 5300, Bulgaria

Location

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

Pleven, Pleven Province, 5800, Bulgaria

Location

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

Plovdiv, Plovdiv Province, 4002, Bulgaria

Location

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

Plovdiv, Plovdiv Province, 4003, Bulgaria

Location

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

Rousse, Ruse Province, 7000, Bulgaria

Location

Multiprofile Hospital for Active Treatment - Shumen, Department of Urology

Shumen, Shumen Province, 9700, Bulgaria

Location

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

Sofia, Sofia-Grad, 1606, Bulgaria

Location

West Tallinn Central Hospital Ltd., Department of Urology

Tallinn, Harju, 10617, Estonia

Location

North Estonia Medical Centre Foundation Ltd.

Tallinn, Harju, 13419, Estonia

Location

East Viru Central Hospital, Surgery Clinic

Kohtla-Järve, Ida-Virumaa, 31025, Estonia

Location

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

Tartu, Tartu County, 50406, Estonia

Location

LLC "Todua Clinic"

Tbilisi, 0119, Georgia

Location

Geo Hospitals LLC

Tbilisi, 0144, Georgia

Location

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

Tbilisi, 0144, Georgia

Location

LTD MMT Hospital

Tbilisi, 0159, Georgia

Location

JSC Jerarsi

Tbilisi, 0167, Georgia

Location

Daugavpils Regional Hospital, Urology Department

Daugavpils, LV-5417, Latvia

Location

Liepajas Regional Hospital, Urology Department

Liepāja, LV-3414, Latvia

Location

P. Stradins Clinical University Hospital, Center for Urology

Riga, LV-1002, Latvia

Location

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

Riga, LV-1079, Latvia

Location

Bihor County Emergency Clinical Hospital

Oradea, Bihor County, 410169, Romania

Location

Brasov County Emergency Clinical Hospital, Department of Urology

Brasov, Brașov County, 500157, Romania

Location

Craiova County Emergency Clinical Hospital, Department of Urology

Craiova, Dolj, 200642, Romania

Location

SC Clinica Polisano Hospital

Sibiu, Sibiu County, 550253, Romania

Location

Colentina Clinical Hospital, Department of Urology

Bucharest, 020125, Romania

Location

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

Bucharest, 050659, Romania

Location

University Hospital Virgen de la Victoria

Málaga, Andalusia, 29010, Spain

Location

Puigvert Foundation

Barcelona, Catalonia, 08025, Spain

Location

University Hospital Foundation Jimenez Diaz

Madrid, Madrid, 28040, Spain

Location

University Hospital 12 de Octubre

Madrid, Madrid, 28041, Spain

Location

La Paz University Hospital

Madrid, Madrid, 28046, Spain

Location

MeSH Terms

Conditions

Urinary Bladder Neoplasms; Carcinoma, Transitional Cell; Non-Muscle Invasive Bladder Neoplasms

Interventions

Mitomycin

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Mitomycins; Indolequinones; Quinones; Organic Chemicals; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Sebastian Mirkin, MD

  UroGen Pharma

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2024
• First Posted: March 26, 2024
• Study Start: August 29, 2024
• Primary Completion: September 11, 2025
• Study Completion (Estimated): September 1, 2026
• Last Updated: January 15, 2026

Record last verified: 2026-01

Locations

RO (6); ES (5); BU (7); US (17); LA (4); GE (5)