A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer

September 2, 2026 updated by: UroGen Pharma Ltd.

A Phase 3, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of UGN-104, a Novel Formulation of UGN-101, for the Treatment of Patients With Low-grade Upper Tract Urothelial Cancer (LG-UTUC)

This study will evaluate the efficacy and safety of UGN-104, a new formulation of UGN-101 (approved in the United States and Israel as JELMYTO [mitomycin] for pyelocalyceal solution), instilled in the upper urinary tract (UUT) of patients with low-grade upper tract urothelial cancer (LG-UTUC).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Eligible patients will be treated with UGN-104 once weekly for 6 weeks (a total of 6 doses).

Efficacy will be assessed by the complete response rate (CRR) at the Primary Disease Evaluation (PDE) Visit (approximately 3 months after the first instillation). Response will be determined based on central laboratory interpretation of UUT wash urine cytology and visual evaluation (ureteroscopy). Biopsy of any remaining or new lesions will be performed where technically feasible and the central laboratory histopathology assessment will also be used in the response determination, if applicable.

Patients who have a complete response (CR) at the PDE Visit, defined as having no detectable disease (NDD), will enter the Follow-up Period of the study. During the Follow-up Period, patients may receive UGN-104 as maintenance treatment at the discretion of the investigator. The maintenance regimen is once monthly for 11 months (a total of 11 doses) and should start as soon as possible (no longer than 3 weeks) after CR is determined at the PDE Visit.

All patients in the Follow-up Period, regardless of whether they receive maintenance treatment, will return to the clinic every 3 months for evaluation of response and will remain on study for 12 months after the PDE Visit or until disease recurrence, disease progression, or death, whichever occurs first.

Patients who have a non-complete response (NCR) at the PDE Visit will be considered to have completed the study.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
70

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Bulgaria
      • Haskovo, Bulgaria, 6300
        • Recruiting
        • MHAT Haskovo AD
        • Contact:
        • Principal Investigator:
          • Ivan Nikolov
      • Sofia, Bulgaria, 1000
        • Recruiting
        • University Multiprofile Hospital for Active Treatment and Emergency Medicine "N.I. Pirogov," Sofia, Clinic of Urology
        • Contact:
        • Principal Investigator:
          • Boris Mladenov
    • Plovdiv
      • Plovdiv, Plovdiv, Bulgaria, 4002
        • Recruiting
        • University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv
        • Contact:
        • Principal Investigator:
          • Petar Antonov, MD
    • Shumen
      • Shumen, Shumen, Bulgaria, 9700
        • Recruiting
        • Multiprofile Hospital for Active Treatment - Shumen, Department of Urology
        • Contact:
        • Principal Investigator:
          • Dobri Marchev, MD
  • Georgia
      • Tbilisi, Georgia, 0144
        • Recruiting
        • LTD "Tbilisi State Medical University and Ingorokva High Medical Technology University Clinic"
        • Contact:
        • Principal Investigator:
          • Shalva Chovelidze
      • Tbilisi, Georgia, 0112
        • Recruiting
        • LTD "Israel-Georgian Medical Research Clinic "Healthycore"
        • Principal Investigator:
          • Ekaterine Arkania
        • Contact:
      • Tbilisi, Georgia, 0137
        • Recruiting
        • JSC "Vian"-I.Bokeria University Hospital
        • Contact:
        • Principal Investigator:
          • Koba Kiknavelidze
      • Tbilisi, Georgia, 0144
        • Recruiting
        • LTD "Geo Hospitals"
        • Contact:
        • Principal Investigator:
          • Giorgia Adeishvili
    • Imereti
      • Kutaisi, Imereti, Georgia
        • Recruiting
        • LTD West Regional Center of Modern Medical Technologies
        • Contact:
        • Principal Investigator:
          • Merab Bzikadze, MD
    • K'alak'i T'bilisi
      • Tbilisi, K'alak'i T'bilisi, Georgia
        • Recruiting
        • Guram Karazanashvili Robotic Center LLC
        • Contact:
        • Principal Investigator:
          • Guram Karazanashvili, MD
      • Tbilisi, K'alak'i T'bilisi, Georgia
        • Recruiting
        • JSC Jerarsi
        • Contact:
        • Principal Investigator:
          • Alexandre Khuskivadze, MD
  • Hungary
      • Debrecen, Hungary, 4032
        • Recruiting
        • University of Debrecen Clinical Center, Department of Urology
        • Contact:
        • Principal Investigator:
          • Tibor Flasko, MD
    • Budapest
      • Budapest, Budapest, Hungary
        • Completed
        • Semmelweis University, Department of Urology
  • Israel
      • Haifa, Israel, 3436212
        • Recruiting
        • Lady Davis Carmel Medical Center
        • Contact:
        • Principal Investigator:
          • Yuval Freifeld
      • Petah Tikva, Israel, 4937211
        • Recruiting
        • Rabin Medical Center - Hasharon hospital
        • Contact:
        • Principal Investigator:
          • David Lifshitz
      • Ẕerifin, Israel, 70500
        • Recruiting
        • Assaf Harofeh hospital, Shamir Medical Center
        • Contact:
        • Principal Investigator:
          • Amnon Zisman
    • Israel
      • Kefar Sava, Israel, Israel, 4428164
        • Recruiting
        • Meir Medical Center, Department of Urology
        • Contact:
        • Principal Investigator:
          • Omri Schwarztuch Gildor
      • Ramat Gan, Israel, Israel, 5262000
        • Recruiting
        • Sheba medical center
        • Principal Investigator:
          • Nir Kleinmann
        • Contact:
  • Latvia
    • Liepaja
      • Liepāja, Liepaja, Latvia, LV-3414
        • Recruiting
        • Liepajas Regional Hospital, Kurzeme Urology Center
        • Contact:
        • Principal Investigator:
          • Dzintra Litavniece, MD
    • Riga
      • Riga, Riga, Latvia, LV-1079
        • Recruiting
        • LLC Riga East University Hospital, Clinic of Urology and Oncologic Urology
        • Contact:
        • Principal Investigator:
          • Vilnis Lietuvietis, MD
      • Riga, Riga, Latvia
        • Not yet recruiting
        • P. Stradins Clinical University Hospital, Center for Urology
        • Contact:
        • Principal Investigator:
          • Egils Vjaters, MD
  • Poland
      • Bydgoszcz, Poland, 85-048
        • Recruiting
        • IN-VIVO Limited Liability Company
        • Principal Investigator:
          • Piotr Piasecki
        • Contact:
      • Katowice, Poland, 40-073
        • Recruiting
        • NZOZ Silesian Urology Hospital
        • Contact:
        • Principal Investigator:
          • Igal Mor
      • Lublin, Poland, 20-090
        • Recruiting
        • St. John of Dukla Oncology Center of Lublin Land
        • Principal Investigator:
          • Robert Klijer, MD
        • Contact:
      • Olsztyn, Poland, 10-228
        • Recruiting
        • Clinical Hospital of the Ministry of Internal Affairs and Administration with Warmia and Mazury Oncology Centre in Olsztyn
        • Contact:
        • Principal Investigator:
          • Roman Sosnowski, MD
      • Szczecin, Poland, 70-111
        • Recruiting
        • Independent Public Teaching Hospital #2
        • Contact:
        • Principal Investigator:
          • Marcin Slojewski, MD
      • Wroclaw, Poland, 50-556
        • Recruiting
        • Jan Mikulicz-Radecki University Clinical Hospital in Wroclaw
        • Contact:
        • Principal Investigator:
          • Anna Kołodziej
    • Opole Voivodeship
      • Opole, Opole Voivodeship, Poland, 45-401
        • Recruiting
        • Opole University Clinical Hospital (USK Opole)
        • Contact:
        • Principal Investigator:
          • Marcin Zyczkowski, MD
    • Pomeranian
      • Gdansk, Pomeranian, Poland
        • Completed
        • University Clinical Center in Gdansk, Department of urology
  • Romania
      • Cluj-Napoca, Romania, 400139
        • Recruiting
        • Municipal Clinical Hospital Cluj-Napoca
        • Contact:
        • Principal Investigator:
          • Ramona Doina Matei
      • Iași, Romania, 700483
        • Recruiting
        • Regional Institute of Oncology Iasi
        • Contact:
        • Principal Investigator:
          • Teodora Alexa Stratulat
      • Oradea, Romania, 410169
        • Recruiting
        • Bihor County Emergency Clinical Hospital
        • Contact:
        • Principal Investigator:
          • Mihail Claudius Berechet
      • Timișoara, Romania, 300723
        • Recruiting
        • Timisoara County Clinical Emergency Hospital Pius Brinzeu
        • Contact:
        • Principal Investigator:
          • Razvan Tiberiu Bardan
    • Bucharest
      • Bucharest, Bucharest, Romania
        • Recruiting
        • "Dr. Carol Davila" Nephrology Clinical Hospital Bucharest
        • Contact:
        • Principal Investigator:
          • Marius Tudor Radu, MD
      • Bucharest, Bucharest, Romania
        • Recruiting
        • Delta Health Care S.R.L.
        • Contact:
        • Principal Investigator:
          • Dragos Razvan Multescu, MD
  • Spain
      • Cadiz, Spain, 11009
        • Recruiting
        • Hospital Universitario Puerta del Mar
        • Contact:
        • Principal Investigator:
          • Jesús Rosety Rodriguez
      • Córdoba, Spain, 14004
        • Recruiting
        • Hospital Universitario Reina Sofia
        • Contact:
        • Principal Investigator:
          • Juan Manuel Rubio Galisteo
      • Murcia, Spain, 30008
        • Recruiting
        • Hospital General Universitario Morales Meseguer
        • Contact:
        • Principal Investigator:
          • Tomás Fernández Aparicio
      • Seville, Spain, 41009
        • Recruiting
        • Hospital Universitario Virgen Macarena
        • Contact:
        • Principal Investigator:
          • Carlos González Cáliz
      • Valencia, Spain, 46010
        • Recruiting
        • Hospital Clínico Universitario de Valencia
        • Contact:
        • Principal Investigator:
          • Jose María Martinez Jabaloyas
    • Catalonia
      • Barcelona, Catalonia, Spain, 08025
        • Recruiting
        • Puigvert Foundation
        • Contact:
        • Principal Investigator:
          • Alberto Breda, MD
    • Galicia
      • A Coruña, Galicia, Spain
        • Recruiting
        • University Hospital Complex A Coruna (CHUAC), Department of Urology
        • Contact:
        • Principal Investigator:
          • Dario Vazquez-Martul
    • Madrid
      • Madrid, Madrid, Spain
        • Recruiting
        • University Hospital Ramon y Cajal
        • Contact:
        • Principal Investigator:
          • Victoria Gomez Dos Santos, MD
      • Madrid, Madrid, Spain, 28006
        • Recruiting
        • University Hospital de La Princesa
        • Contact:
        • Principal Investigator:
          • Luis A. San Jose Manso, MD
      • Madrid, Madrid, Spain, 28040
        • Recruiting
        • University Hospital Clinical San Carlos, Department of Urology
        • Contact:
        • Principal Investigator:
          • Maria Isabel Galante Romo, MD
  • United States
    • Alabama
      • Homewood, Alabama, United States, 35209
        • Recruiting
        • Urology Centers of Alabama
        • Principal Investigator:
          • Michael Eric Brewer
        • Contact:
    • Arizona
      • Chandler, Arizona, United States, 85225
        • Recruiting
        • Arizona State Urological Research Institute, LLC ("ASURI")
        • Contact:
        • Principal Investigator:
          • Pratik Patel
      • Queen Creek, Arizona, United States, 85140
        • Recruiting
        • East Valley Urology Center of Arizona
        • Principal Investigator:
          • Harpreet Wadhwa, MD
        • Contact:
    • Arkansas
      • Arkansas City, Arkansas, United States, 72205
        • Recruiting
        • University of Arkansas for Medical Sciences
        • Principal Investigator:
          • Marcelo Bigarella, MD
        • Contact:
    • California
      • Bakersfield, California, United States, 93301
        • Recruiting
        • Michael G Oefelein Clinical Trials
        • Contact:
        • Principal Investigator:
          • Michael Oefelein
      • Orange, California, United States, 92868
        • Recruiting
        • UC Irvine Health. Chao Family Comprehensive Cancer Center
        • Principal Investigator:
          • Edward Uchio
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • MedStar Washington Hospital Center
        • Principal Investigator:
          • Lambros Stamatakis, MD
        • Contact:
    • Florida
      • Aventura, Florida, United States, 33180
        • Recruiting
        • Bioresearch Partner - Aventura
        • Contact:
        • Principal Investigator:
          • Angelo Gousse
      • Gainesville, Florida, United States, 32610
        • Recruiting
        • University of Florida Health (UF Health), Cancer Center
        • Principal Investigator:
          • Padraic O Malley
        • Contact:
      • Hialeah, Florida, United States, 33013
        • Recruiting
        • Bioresearch Partner - Hialeah Hospital
        • Principal Investigator:
          • Luis Rangel
        • Contact:
      • Margate, Florida, United States, 33063
        • Recruiting
        • D&H National Research Centers - Cancer Research Center, LLC
        • Principal Investigator:
          • Emilio Araujo-Mino, MD
        • Contact:
    • Iowa
      • Clive, Iowa, United States, 50325
        • Recruiting
        • Urology Center of Iowa Research
        • Principal Investigator:
          • Brian Gallagher
        • Contact:
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Completed
        • The Urology Center, P.C.
    • New Jersey
      • New Brunswick, New Jersey, United States, 08903
        • Recruiting
        • Rutgers Cancer Institute of New Jersey
        • Principal Investigator:
          • Saum Ghodoussipour, MD
        • Contact:
    • New York
      • Rochester, New York, United States, 14642
        • Not yet recruiting
        • University of Rochester Medical Center
        • Contact:
        • Principal Investigator:
          • Phillip Rappold, MD
      • The Bronx, New York, United States, 10461
        • Recruiting
        • Montefiore Medical Center - Bronx
        • Principal Investigator:
          • Alexander Small, MD
        • Contact:
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Recruiting
        • Urology Specialists of the Carolinas, PLLC
        • Principal Investigator:
          • Jeremy Hubbard, MD
        • Contact:
      • Greensboro, North Carolina, United States, 27403
        • Recruiting
        • Urology Specialists of the Carolinas - Greensboro
        • Principal Investigator:
          • Jeremy Hubbard, MD
        • Contact:
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Recruiting
        • University of Cincinnati Medical Center (UCMC)
        • Contact:
        • Principal Investigator:
          • Alberto Martini
      • Columbus, Ohio, United States, 43210
        • Recruiting
        • The James Cancer Hospital and Solove Research Institute
        • Contact:
        • Principal Investigator:
          • Ahmad Shabsigh, MD
    • Oklahoma
      • Tulsa, Oklahoma, United States, 74146-5830
        • Recruiting
        • Urologic Specialists of Oklahoma
        • Principal Investigator:
          • John Forrest, MD
        • Contact:
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Recruiting
        • Penn State Milton S Hershey Medical Center
        • Principal Investigator:
          • Jay Raman
        • Contact:
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina
        • Contact:
        • Principal Investigator:
          • Matvey Tsivian
      • Myrtle Beach, South Carolina, United States, 29572
        • Recruiting
        • Carolina Urologic Research Center, LLC
        • Contact:
        • Principal Investigator:
          • Abhishek Srivastava
      • North Charleston, South Carolina, United States, 29406
        • Recruiting
        • Low county Urology Clinics
        • Contact:
        • Principal Investigator:
          • Justin Ellett
    • Texas
      • Amarillo, Texas, United States, 79106
        • Completed
        • Amarillo Urology Research
      • Arlington, Texas, United States, 76017
        • Recruiting
        • UPNT Research Institute, LLC
        • Principal Investigator:
          • Harrison Abrahams
        • Contact:
      • Austin, Texas, United States, 78705
        • Recruiting
        • Texas Urology Specialists-Austin
        • Contact:
        • Principal Investigator:
          • Kyle Keyes
      • Dallas, Texas, United States, 75390
        • Completed
        • The University of Texas Southwestern Medical Center
      • Houston, Texas, United States, 77027
        • Recruiting
        • Houston Metro Urology (HMU) - Southwest Location
        • Principal Investigator:
          • Zvi Schiffman, MD
        • Contact:
      • Houston, Texas, United States, 77090
        • Recruiting
        • Lumi Research - Houston
        • Contact:
        • Principal Investigator:
          • Pullin Pandya, MD
      • San Antonio, Texas, United States, 78229
      • The Woodlands, Texas, United States, 77380
        • Recruiting
        • Renovatio Clinical - The Woodlands Research Center
        • Principal Investigator:
          • Mahran Shoukier, MD
        • Contact:
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • Recruiting
        • University of Virginia Comprehensive Cancer Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Tracy Krupski

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Provide written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and protocol.

  2. Naive or recurrent patients with LG, non-invasive UTUC in the pyelocalyceal system, with the following disease characteristics:

    1. At least 1 measurable papillary LG tumor, evaluated visually, ≤ 15 mm. The largest lesion should not exceed 15 mm. Debulking to ≤ 15 mm is permitted.
    2. Biopsy taken from at least 1 tumor located above the ureteropelvic junction revealing LG urothelial carcinoma (UC). Patients who have been biopsied within 8 weeks before Screening for this study and shown to have LG UC may have these historical biopsies used for enrollment into the study and do not require repeat biopsy during Screening.
    3. Patient should have at least 1 remaining papillary LG tumor evaluated visually with a diameter of at least 5 mm post-biopsy.
    4. Wash urine cytology sampled from the pyelocalyceal system documenting the absence of high-grade (HG) UC, diagnosed not more than 8 weeks before Screening.
    5. Patients with bilateral LG-UTUC may be enrolled if at least 1 side meets the inclusion criteria for the study and if the other kidney does not require further treatments. (The disease in the other kidney must be completely ablated before receiving treatment in the study.) Note: If both upper tracts meet inclusion criteria, the treating urologist in consultation with the sponsor's medical monitor can decide which side to treat in the study. The pyelocalyceal system not under study must be free of cancer before the first instillation on the side to be treated in the study.
  3. Patients with Eastern Cooperative Oncology Group (ECOG) performance status < 3 (with Karnofsky > 40).
  4. Patients with life expectancy > 24 months at time of Screening.

  5. Patients must have adequate organ and bone marrow function as determined by the following routine laboratory tests:

    1. Leukocytes ≥ 3,000/μL (≥ 3 × 10^9/L).
    2. Absolute neutrophil count ≥ 1,500/μL (≥ 1.5 × 10^9/L).
    3. Platelets ≥ 100,000/μL (≥ 100 × 10^9/L).
    4. Hemoglobin ≥ 9.0 g/dL.
    5. Total bilirubin ≤ 1.5 × upper limit of normal (ULN).
    6. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN.
    7. Alkaline phosphatase ≤ 2.5 × ULN.
    8. Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min.
  6. Patient has no active urinary tract infection (UTI) as confirmed by urine culture or urinalysis. Note: In case of a symptomatic UTI the patient will be treated with antibiotics and the instillation will be postponed until resolution. In the case of asymptomatic bacteriuria, the use of prophylactic antibiotics and postponement of treatment is at the discretion of the investigator.
  7. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for clinical study participants.

Female patients of childbearing potential (defined as premenopausal women who have not been sterilized) and male patients with female partners of childbearing potential must agree to use 2 acceptable forms of effective contraception from enrollment through 6 months post treatment. Sexually active male patients must agree to use a condom during intercourse for at least 48 hours after each instillation.

Acceptable methods of birth control considered to have a low failure rate (ie, less than 1% per year) when used consistently and correctly include implants, injectable, combined (estrogen/progesterone) oral contraceptives, intrauterine devices (only hormonal), condoms with spermicide, sexual abstinence* or vasectomized partner.

* Sexual abstinence is defined as refraining from intercourse from enrollment through 6 months post treatment. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is NOT an acceptable method of contraception.

Exclusion Criteria:

  1. UC specific exclusions:

    1. Patient received Bacillus Calmette-Guérin (BCG) treatment for UC during the 6 months before enrollment.
    2. The patient has untreated concurrent UC in locations other than the target area (unless treated and resolved during Screening); ie, ureteral and lower urinary tract tumors must be completely ablated before entry.
    3. Patient has a history of carcinoma in situ (CIS) in the urinary tract.
    4. Patient has a history of invasive UC in the past 5 years.
    5. Patient has a history of HG papillary UC in the urinary tract in the past 2 years.
  2. Patient is actively being treated or intends to be treated with systemic chemotherapy during the duration of the study.

  3. Any other malignancy diagnosed within 2 years before enrollment with the exception of:

    1. Basal or squamous cell skin cancers.
    2. Noninvasive cancer of the cervix.
    3. Any other cancer deemed to be of low risk for progression or patient morbidity during the study period in the opinion of the investigator.
  4. Patient with urinary obstruction such that there is an inability to deliver the study treatment to the pyelocalyceal system either via ureteral catheter (retrograde administration) or nephrostomy tube (antegrade administration).
  5. Known allergy or sensitivity to any component of the study treatment (including excipients) that in the investigator's opinion cannot be readily managed.
  6. Patient has an intractable bleeding disorder (eg, coagulation factors deficiencies, Von Willebrand disease).
  7. Patient is currently receiving any other investigational product, has participated in a research protocol involving administration of an investigational product in the past 30 days, or plans to participate in a research protocol involving administration of an investigational product during study conduct.
  8. Patient was previously treated with JELMYTO (product code UGN-101) for UTUC.
  9. Women who are pregnant (positive urine or serum pregnancy test), planning to become pregnant during the study period, or who are breastfeeding are not eligible to enroll.
  10. Patient has any other medical or mental condition(s) that make(s) his/her participation in the study unadvisable in the opinion of the investigator.
  11. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: UGN-104

Patients will receive UGN-104 once weekly for 6 weeks (a total of 6 doses). The dose of UGN-104 to be instilled is 4 mg mitomycin per 1 mL sterile hydrogel via ureteral catheter (retrograde administration) or nephrostomy tube (antegrade administration).

Patients who have a CR at the PDE Visit will enter the Follow-up Period and may receive UGN-104 as maintenance treatment once monthly for 11 months (a total of 11 doses) at the discretion of the investigator.
Drug: UGN-104
UGN-104 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-104 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 UUT.
Other Names:
  • UGN-104 (mitomycin) for pyelocalyceal solution

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Complete response rate (CRR)
Time Frame: 3 months
CRR is defined as the proportion of patients who achieved CR at the PDE Visit.
3 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Duration of response (DOR)
Time Frame: 12 months
DOR is defined as the time from the date of evidence of CR at the PDE Visit to the earliest date of recurrence, progression, or death due to any cause, whichever occurred first.
12 months
Durable complete response (DCR) rate
Time Frame: 12 months
DCR rate at scheduled disease assessment time points, defined as the proportion of patients who achieved CR at the PDE Visit and maintained CR (ie, no detectable disease) up to that particular follow-up disease assessment.
12 months
Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, TEAEs of special interest, and abnormal clinical laboratory test results (hematology and serum chemistry)
Time Frame: 15 months
The number of patients with each type of event will be summarized.
15 months
Mitomycin plasma concentrations
Time Frame: 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin plasma concentrations will be assessed in a subset of patients treated with UGN-104.
0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin maximum plasma concentration (Cmax)
Time Frame: 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin Cmax will be assessed in a subset of patients treated with UGN-104.
0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin time to maximum plasma concentration (tmax)
Time Frame: 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin tmax will be assessed in a subset of patients treated with UGN-104.
0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin area under the concentration-time curve (AUC)
Time Frame: 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin AUC will be assessed in a subset of patients treated with UGN-104.
0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin terminal half-life (t1/2)
Time Frame: 0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104
Mitomycin t1/2 will be assessed in a subset of patients treated with UGN-104.
0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
UroGen Pharma Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Derrick McKinley, MD, UroGen Pharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 6, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 1, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 8, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 8, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 14, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 3, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 2, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • UT002
  • 2024-519343-15-00 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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