Home
Clinical Trials
A Phase 3 Single-arm Study of...

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

Last Updated: 2026-01-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-08-06

Study Completion Date

2027-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Efficacy and Safety of UGN-101 in Recurrent Patients

NCT04006691

Carcinoma Transitional Cell Carcinoma Transitional Cell Cancer of the Renal Pelvis and Ureter

WITHDRAWN PHASE3

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

NCT06331299

Bladder Cancer Urothelial Carcinoma Urothelial Carcinoma Bladder

ACTIVE_NOT_RECRUITING PHASE3

A Phase 3 Single-Arm Study of UGN-102 for Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

NCT05243550

Bladder Cancer Urothelial Carcinoma Urothelial Carcinoma Bladder

ACTIVE_NOT_RECRUITING PHASE3

uTRACT Jelmyto Registry: A Registry of Patients With Upper Tract Urothelial Cancer (UTUC) Treated With Jelmyto

NCT05874921

Urothelial Carcinoma Urothelial Carcinoma Recurrent Urothelial Carcinoma Ureter +5 more

RECRUITING

A Phase 2b Study of UGN-102 for Low Grade Intermediate Risk Non-Muscle-Invasive Bladder Cancer

NCT03558503

Bladder Cancer Urothelial Carcinoma Urothelial Carcinoma Bladder

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Upper Urinary Tract Urothelial Carcinoma Urothelial Carcinoma

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

UGN-104

Intervention Type DRUG

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

UGN-104

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

UGN-104 (mitomycin) for pyelocalyceal solution

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers