Actively Recruiting
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)
Led by UroGen Pharma Ltd. · Updated on 2026-04-29
70
Participants Needed
52
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of UGN-104, a new formulation of UGN-101 (also known as JELMYTO), for treating patients with low-grade upper tract urothelial cancer (LG-UTUC). This phase 3, single-arm, multicenter study focuses on patients with measurable papillary tumors in the upper urinary tract. The study aims to determine how well UGN-104 works by measuring the complete response rate about 3 months after starting treatment. Participants will receive UGN-104 once weekly for 6 weeks, with a dose of 4 mg mitomycin in a sterile hydrogel instilled into the upper urinary tract either retrograde via ureteral catheter or antegrade via nephrostomy tube. Patients who achieve a complete response at the primary disease evaluation visit may enter a follow-up period, where they could receive monthly maintenance doses of UGN-104 for 11 months, starting within 3 weeks of response confirmation. Those without complete response will complete the study after the initial treatment phase. During the study, participants will have regular evaluations including urine cytology and ureteroscopy to assess tumor response, with biopsies performed if needed. Follow-up visits occur every 3 months for up to 12 months after the primary evaluation or until disease recurrence, progression, or death. Researchers will also monitor treatment-emergent side effects and measure mitomycin levels in the blood to understand how the drug behaves in the body. The overall goal is to assess both the duration and durability of response while ensuring safety over a 15-month period.
CONDITIONS
Brief Title
A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide written informed consent.
- Have low-grade, non-invasive upper tract urothelial cancer with at least one measurable papillary tumor 15 mm or smaller.
- Have a biopsy confirming low-grade urothelial carcinoma above the ureteropelvic junction.
- Have at least one remaining papillary tumor 5 mm or larger after biopsy.
- Have urine cytology showing no high-grade urothelial carcinoma within 8 weeks before screening.
- If bilateral disease, one side must meet criteria and the other kidney must be cancer-free.
- Have ECOG performance status less than 3 and Karnofsky score above 40.
- Have life expectancy over 24 months.
- Have adequate organ and bone marrow function based on laboratory tests.
- Have no active urinary tract infection.
- Use effective contraception if of childbearing potential, following study guidelines.
You will not qualify if you...
- Received BCG treatment for urothelial cancer in the past 6 months.
- Have untreated urothelial cancer outside the target area.
- History of carcinoma in situ in the urinary tract.
- History of invasive urothelial carcinoma within the past 5 years.
- History of high-grade papillary urothelial carcinoma within the past 2 years.
- Currently receiving or planning systemic chemotherapy during the study.
- Other recent malignancies within 2 years, except certain low-risk cancers.
- Urinary obstruction preventing study drug delivery.
- Allergy or sensitivity to study treatment components.
- Intractable bleeding disorders.
- Participation in other investigational studies within 30 days or plans to do so during this study.
- Previous treatment with JELMYTO (UGN-101).
- Pregnant, planning pregnancy, or breastfeeding.
- Other medical or mental conditions that make participation unsafe.
- Legal commitment to an institution where applicable by local regulations.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 6 weeks
Participants receive UGN-104 once weekly for 6 weeks (a total of 6 doses) via ureteral catheter or nephrostomy tube.
6 weekly visits (in-person) for dosing
Duration - Approximately 3 months after treatment start
Approximately 3 months after the first instillation, participants undergo evaluation to assess complete response using urine cytology, ureteroscopy, and biopsy if needed.
1 visit (in-person)
Duration - Up to 12 months after primary disease evaluation
Participants with complete response at the evaluation may receive maintenance UGN-104 once monthly for 11 months at the investigator's discretion. All participants in follow-up return every 3 months for evaluation of response and remain on study for 12 months after the evaluation or until disease recurrence, progression, or death.
Monthly visits for maintenance dosing and quarterly visits for evaluation during follow-up
Trial Site Locations
Total: 52 locations
1
Urology Centers of Alabama
Homewood, Alabama, United States, 35209
Actively Recruiting
2
Arizona State Urological Research Institute, LLC ("ASURI")
Chandler, Arizona, United States, 85225
Actively Recruiting
3
1155 E. Combs Rd, Suite 105, Queen Creek, AZ, 85140, USA
Queen Creek, Arizona, United States, 85140
Actively Recruiting
4
Michael G Oefelein Clinical Trials
Bakersfield, California, United States, 93301
Actively Recruiting
5
University of California, Irvine
Orange, California, United States, 92868
Actively Recruiting
6
BioResearch Partners Aventura
Aventura, Florida, United States, 33180
Actively Recruiting
7
UF Health Cancer Center University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
8
BioResearch Partners Hialeah
Hialeah, Florida, United States, 33013
Actively Recruiting
9
D&H National Research Centers - Cancer Research Center, LLC
Margate, Florida, United States, 33063
Actively Recruiting
10
Urology Center of Iowa Research
Clive, Iowa, United States, 50325
Actively Recruiting
11
The Urology Center, P.C.
Omaha, Nebraska, United States, 68114
Actively Recruiting
12
Montefiore Medical Center - Bronx
The Bronx, New York, United States, 10461
Actively Recruiting
13
University of Cincinnati Medical Center (UCMC)
Cincinnati, Ohio, United States, 45219
Actively Recruiting
14
The James Cancer Hospital and Solove Research Institute
Columbus, Ohio, United States, 43210
Actively Recruiting
15
Urologic Specialists of Oklahoma
Tulsa, Oklahoma, United States, 74146-5830
Actively Recruiting
16
Penn State Milton S Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
17
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
18
Carolina Urologic Research Center, LLC
Myrtle Beach, South Carolina, United States, 29572
Actively Recruiting
19
Low county Urology Clinics
North Charleston, South Carolina, United States, 29406
Actively Recruiting
20
Amarillo Urology Research
Amarillo, Texas, United States, 79106
Actively Recruiting
21
UPNT Research Institute, LLC
Arlington, Texas, United States, 76017
Actively Recruiting
22
Texas Urology Specialists-Austin
Austin, Texas, United States, 78705
Actively Recruiting
23
University of Virginia Comprehensive Cancer Center
Charlottesville, Virginia, United States, 22908
Actively Recruiting
24
MHAT Haskovo AD
Haskovo, Bulgaria, 6300
Actively Recruiting
25
University Multiprofile Hospital for Active Treatment and Emergency Medicine "N.I. Pirogov," Sofia, Clinic of Urology
Sofia, Bulgaria, 1000
Actively Recruiting
26
LTD "Israel-Georgian Medical Research Clinic "Healthycore"
Tbilisi, Georgia, 0112
Actively Recruiting
27
JSC "Vian"-I.Bokeria University Hospital
Tbilisi, Georgia, 0137
Actively Recruiting
28
LTD "Geo Hospitals"
Tbilisi, Georgia, 0144
Actively Recruiting
29
LTD "Tbilisi State Medical University and Ingorokva High Medical Technology University Clinic"
Tbilisi, Georgia, 0144
Actively Recruiting
30
University of Debrecen Clinical Center, Department of Urology
Debrecen, Hungary, 4032
Actively Recruiting
31
Carmel Medical Center
Haifa, Israel, 3436212
Actively Recruiting
32
Meir Medical Center, Department of Urology
Kefar Sava, Israel, 4428164
Actively Recruiting
33
Rabin Medical Center - Hasharon hospital
Petah Tikva, Israel, 4937211
Actively Recruiting
34
Sheba Medical Center
Ramat Gan, Israel, 5262000
Actively Recruiting
35
Sheba Medical Center
Ramat Gan, Israel, 5262000
Actively Recruiting
36
Assaf Harofeh hospital, Shamir Medical Center
Ẕerifin, Israel, 70500
Actively Recruiting
37
IN-VIVO Limited Liability Company
Bydgoszcz, Poland, 85-048
Actively Recruiting
38
NZOZ Silesian Urology Hospital
Katowice, Poland, 40-073
Actively Recruiting
39
St. John of Dukla Oncology Center of Lublin Land
Lublin, Poland, 20-090
Actively Recruiting
40
Clinical Hospital of the Ministry of Internal Affairs and Administration with Warmia and Mazury Oncology Centre in Olsztyn
Olsztyn, Poland, 10-228
Actively Recruiting
41
Independent Public Teaching Hospital #2
Szczecin, Poland, 70-111
Actively Recruiting
42
Jan Mikulicz-Radecki University Clinical Hospital in Wroclaw
Wroclaw, Poland, 50-556
Actively Recruiting
43
Municipal Clinical Hospital Cluj-Napoca
Cluj-Napoca, Romania, 400139
Actively Recruiting
44
Regional Institute of Oncology Iasi
Iași, Romania, 700483
Actively Recruiting
45
Bihor County Emergency Clinical Hospital
Oradea, Romania, 410169
Actively Recruiting
46
Timisoara County Clinical Emergency Hospital Pius Brinzeu
Timișoara, Romania, 300723
Actively Recruiting
47
15006
A Coruña, Galicia, Spain
Actively Recruiting
48
Hospital Universitario Puerta del Mar
Cadiz, Spain, 11009
Actively Recruiting
49
Hospital Universitario Reina Sofía
Córdoba, Spain, 14004
Actively Recruiting
50
Hospital General Universitario Morales Meseguer
Murcia, Spain, 30008
Actively Recruiting
51
Hospital Universitario Virgen Macarena
Seville, Spain, 41009
Actively Recruiting
52
Hospital Clínico Universitario de Valencia
Valencia, Spain, 46010
Actively Recruiting
Research Team
R
Renee Sumlin
Y
Yafit Makmal
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here