Bladder CancerPhase 3UTOPIANCT06331299
A Phase 3 Study of UGN-103 for Treatment of Patients With Low-grade Intermediate-risk Non-muscle Invasive Bladder Cancer
A Phase 3 study testing UGN-103 in people with Bladder Cancer and Urothelial Carcinoma, run by UroGen Pharma Ltd. at 48 sites in 7 countries.
Trial summary
This Phase 3, single-arm, multicenter study was designed to evaluate the efficacy and safety of UGN-103, a novel formulation of UGN-102, instilled in the urinary bladder of patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bladder Cancer, Urothelial Carcinoma, Urothelial Carcinoma Bladder
- What is being tested?
- UGN-103
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 99 participants
- Where
- 48 sitesin 7 countries
- Study dates
- 29 Aug 2024to 15 Sep 2026
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
- Alkaline phosphatase (ALP) ≤ 2.5 × ULN.
- Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min.
- Has an anticipated life expectancy of at least the duration of the trial.
- 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.
Who cannot join
- 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.
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- 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).
- Has participated in a study with an investigational agent or device within 30 days of enrollment.
- Has previously participated in a study in which they received UGN-102.
- 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.
- 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.
- 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.
Trial locations and contacts
48 sites in 7 countries.
48 sites in 7 countries
United States17 sites
Great Lakes Physician dba WNYU
Cheektowaga, New York, 14225
AccuMed Research Associates
Garden City, New York, 11530
Houston Metro Urology (HMU) - Southwest Location
Houston, Texas, 77027
Houston Methodist Hospital
Houston, Texas, 77030
Indiana University Melvin and Bren Simon Cancer Center
Indianapolis, Indiana, 46202
Loma Linda University Medical Center
Loma Linda, California, 92354
Crystal Run Healthcare
Middletown, New York, 10941
Garden State Urology
Morristown, New Jersey, 07962
Carolina Urologic Research Center
Myrtle Beach, South Carolina, 29572
Urology Associates P.C. - Nashville
Nashville, Tennessee, 37209-4035
Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York, New York, 10016
Perlmutter Cancer Center at NYU Langone Hospital - Long Island
New York, New York, 11501
Peachtree Clinical Solutions
Powder Springs, Georgia, 30127
Genesis Research, LLC
San Diego, California, 92123
Spokane Urology, P.S.
Spokane, Washington, 99202
The George Washington University Medical Faculty Associates
Washington D.C., District of Columbia, 20037
Minnesota Urology - Metro Urology - Woodbury
Woodbury, Minnesota, 55125
Bulgaria7 sites
Multiprofile Hospital for Active Treatment "Dr. Tota Venkova", Gabrovo, Department of Urology
Gabrovo, Gabrovo, 5300
University Multiprofile Hospital for Active Treatment "Dr. Georgi Stranski", Pleven, Urology Clinic
Pleven, Pleven Province, 5800
University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv, Clinic of Urology
Plovdiv, Plovdiv Province, 4002
University Multiprofile Hospital for Active Treatment, Plovdiv, Clinic of Urology
Plovdiv, Plovdiv Province, 4003
University Multiprofile Hospital for Active Treatment 'Kanev', Ruse, Department of Urology
Rousse, Ruse Province, 7000
Multiprofile Hospital for Active Treatment - Shumen, Department of Urology
Shumen, Shumen Province, 9700
University Multiprofile Hospital for Active Treatment and Emergency Medicine "N.I. Pirogov", Sofia, Urology Clinic
Sofia, Sofia-Grad, 1606
Romania6 sites
Brasov County Emergency Clinical Hospital, Department of Urology
Brasov, Brașov County, 500157
Colentina Clinical Hospital, Department of Urology
Bucharest, Bucharest, 020125
"Prof. Dr. Th. Burghele" Clinical Hospital, Department of Urology III
Bucharest, Bucharest, 050659
Craiova County Emergency Clinical Hospital, Department of Urology
Craiova, Dolj, 200642
Bihor County Emergency Clinical Hospital
Oradea, Bihor County, 410169
SC Clinica Polisano Hospital
Sibiu, Sibiu County, 550253
Georgia5 sites
LLC "Todua Clinic"
Tbilisi, K'alak'i T'bilisi, 0119
Geo Hospitals LLC
Tbilisi, K'alak'i T'bilisi, 0144
Tbilisi State Medical University and Ingorokva High Medical Technology University Clinic LLC
Tbilisi, K'alak'i T'bilisi, 0144
LTD MMT Hospital
Tbilisi, K'alak'i T'bilisi, 0159
JSC Jerarsi
Tbilisi, K'alak'i T'bilisi, 0167
Spain5 sites
Puigvert Foundation
Barcelona, Catalonia, 08025
University Hospital Foundation Jimenez Diaz
Madrid, Madrid, 28040
University Hospital 12 de Octubre
Madrid, Madrid, 28041
La Paz University Hospital
Madrid, Madrid, 28046
University Hospital Virgen de la Victoria
Málaga, Andalusia, 29010
Estonia4 sites
East Viru Central Hospital, Surgery Clinic
Kohtla-Järve, Ida-Virumaa, 31025
West Tallinn Central Hospital Ltd., Department of Urology
Tallinn, Harju, 10617
North Estonia Medical Centre Foundation Ltd.
Tallinn, Harju, 13419
Tartu University Hospital, Surgery Clinic, Department of Urology and Kidney Transplantation
Tartu, Tartu County, 50406
Latvia4 sites
Daugavpils Regional Hospital, Urology Department
Daugavpils, Daugavpils, LV-5417
Liepajas Regional Hospital, Urology Department
Liepāja, Liepaja, LV-3414
P. Stradins Clinical University Hospital, Center for Urology
Riga, Riga, LV-1002
LLC Riga East University Hospital, Clinic of Urology and Oncologic Urology
Riga, Riga, LV-1079
About this study
Official title: 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 Phase 3, single-arm, multicenter study was designed to evaluate the efficacy and safety of UGN-103, a novel formulation of UGN-102, instilled in the urinary bladder of patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).
Read the detailed description
Eligible patients were treated with UGN-103 once weekly for 6 weeks (a total of 6 doses).
Efficacy was assessed by the complete response rate (CRR) at the 3-month Visit (approximately 3 months after the first instillation). Response was 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 had a complete response (CR) at the 3-month Visit, defined as having no detectable disease (NDD) in the bladder, entered the Follow-up Period of the study. During the Follow-up Period, patients return to the clinic every 3 months for evaluation of response. Patients 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 had a non-complete response (NCR) at the 3-month Visit underwent investigator-designated standard of care (SOC) and had a separate End of Study (EOS) Visit performed.
Study conduct is ongoing and data are summarized through a cutoff date of 18 Mar 2026. As of the data cutoff date, ongoing patients were followed through at least Study Month 9, with the earliest enrolled patients followed through Study Month 15.
- Being tested
- UGN-103
- 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
- Medical subject headings
- Urinary Bladder Neoplasms, Carcinoma, Transitional Cell, Non-Muscle Invasive Bladder Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UGN-103Drug
Also called UGN-103 (mitomycin) for intravesical solution
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.
| Study group | Role | What they receive |
|---|---|---|
| UGN-103 | Experimental | Patients received UGN-103 (75 mg mitomycin) once weekly for 6 weeks (a total of 6 doses). Receives: Drug: UGN-103 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response Rate (CRR)
CRR is defined as the proportion of patients who achieved a complete response (CR) at the 3-month Visit. A patient was considered a CR if there was no detectable disease in the bladder based on visual observation (white light cystoscopy), biopsy of remaining lesions (if applicable), and voiding urine cytology.
Measured: 3 months
Secondary outcomes (7)
Duration of Response (DOR) in Patients Who Achieved CR at the 3-month Visit
DOR is defined as the time from the date of evidence of CR at the 3-month Visit to the earliest date of recurrence or progression or death due to any cause, whichever occurred first.
Measured: Up to 15 months
Durable Complete Response (DCR) Rate in Patients Who Achieved CR at the 3-month Visit
DCR rate at scheduled disease assessment time points, defined as the proportion of patients who achieved CR at the 3-month Visit and maintained CR (ie, no detectable disease) up to that particular follow-up disease assessment.
Measured: Up to 15 months
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs of Special Interest
The number of patients with each type of event is summarized. TEAEs were defined as adverse events (AEs) that started on or after the day of the first instillation of UGN-103 or pre-treatment AEs that worsened during the study.
Measured: Up to 15 months
Number of Participants With Post-baseline Potentially Clinically Significant (PCS) Hematology Values
The number of patients with each type of event is summarized.
Measured: 6 months
Number of Participants With Post-baseline PCS Chemistry Values
The number of patients with each type of event is summarized.
Measured: 6 months
Mitomycin Plasma Concentrations
Mitomycin plasma concentrations were assessed in a subset of patients treated with UGN-103.
Measured: 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)
Mitomycin Cmax was assessed in a subset of patients treated with UGN-103.
Measured: 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-103.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2024
- Study start29 Aug 2024
- Primary completion11 Sep 2025
- Study completion15 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Bladder CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 99 participants (actual)
Sponsor and investigators
- Lead sponsor
- UroGen Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sebastian Mirkin, MD, Study director, UroGen PharmaJoao P Zambon, MD, Study director, UroGen Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06331299
- Sponsor's protocol ID
- BL013
- Acronym
- UTOPIA
- Status last verified
- Oct 2026
- First submitted
- 19 Mar 2024
- Last updated
- 22 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No