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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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

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
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
Are there results?
Yes, posted in the registrySummary in the doctors' view

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.
Show all 19Show fewer
  • 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.
Show all 20Show fewer
  • 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

Other trials in United States

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

Other trials in Bulgaria

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

Other trials in Romania

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

Other trials in Georgia

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

Other trials in Spain

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

Other trials in Estonia

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

Other trials in Latvia

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 groups in NCT06331299
Study groupRoleWhat they receive
UGN-103ExperimentalPatients 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.

  1. 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)

  1. 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

  2. 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

  3. 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

  4. 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

  5. Number of Participants With Post-baseline PCS Chemistry Values

    The number of patients with each type of event is summarized.

    Measured: 6 months

  6. 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.

  7. 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.

  1. First posted26 Mar 2024
  2. Study start29 Aug 2024
  3. Primary completion11 Sep 2025
  4. Study completion15 Sep 2026

Resources

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)
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
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

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.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 48 sites in United States, Bulgaria, Romania, Georgia, Spain and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 28 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Complete Response Rate (CRR) (time frame: 3 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06331299.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.

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