RECRUITING

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

Conditions

Upper Urinary Tract Urothelial Carcinoma Urothelial Carcinoma UTUC Low Grade UTUC Low-grade Upper Tract Urothelial Cancer UGN-104 Mitomycin Nephrostomy Tube LG-UTUC UGN-101

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

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)

Quick Facts

Study Start:2025-05-02

Study Completion:2027-03-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06774131

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.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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 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.	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. 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.

Inclusion Criteria

Exclusion 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 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.

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.
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.

Contacts and Locations

Study Contact

Karrie Hilsinger

CONTACT

609-256-8316

karrie.hilsinger@urogen.com

Yafit Makmal

CONTACT

yafit.makmal@urogen.com

Principal Investigator

Sebastian Mirkin, MD

STUDY_DIRECTOR

UroGen Pharma

Study Locations (Sites)

BioResearch Partners Aventura

Aventura, Florida, 33180

United States

BioResearch Partners Hialeah

Hialeah, Florida, 33013

United States

Penn State Milton S Hershey Medical Center

Hershey, Pennsylvania, 17033

United States

Collaborators and Investigators

Sponsor: UroGen Pharma Ltd.

Sebastian Mirkin, MD, STUDY_DIRECTOR, 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 Registration Dates

Study Start Date2025-05-02

Study Completion Date2027-03-31

Study Record Updates

Study Start Date2025-05-02

Study Completion Date2027-03-31

Terms related to this study

Keywords Provided by Researchers

UTUC
Low Grade UTUC
Low-grade Upper Tract Urothelial Cancer
UGN-104
Mitomycin
Nephrostomy Tube
LG-UTUC
UGN-101

Additional Relevant MeSH Terms

Upper Urinary Tract Urothelial Carcinoma
Urothelial Carcinoma