Recruiting
Phase 3
A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer
Condition(s) studied
Upper Urinary Tract Urothelial CarcinomaUrothelial Carcinoma
Investigational drug(s) / intervention(s)
UGN-104
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.
Study summary
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).
Eligibility
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.
Primary outcome measure(s)
- Complete response rate (CRR) — 3 months
CRR is defined as the proportion of patients who achieved CR at the PDE Visit.
Trial sites (79)
| Facility | City | Region | Status |
| Urology Centers of Alabama |
Homewood |
Alabama |
Recruiting |
| Arizona State Urological Research Institute, LLC ("ASURI") |
Chandler |
Arizona |
Recruiting |
| East Valley Urology Center of Arizona |
Queen Creek |
Arizona |
Recruiting |
| University of Arkansas for Medical Sciences |
Arkansas City |
Arkansas |
Recruiting |
| Michael G Oefelein Clinical Trials |
Bakersfield |
California |
Recruiting |
| UC Irvine Health. Chao Family Comprehensive Cancer Center |
Orange |
California |
Recruiting |
| Medstar Washington Hospital Center |
Washington D.C. |
District of Columbia |
Recruiting |
| Bioresearch Partner - Aventura |
Aventura |
Florida |
Recruiting |
| University of Florida Health (UF Health), Cancer Center |
Gainesville |
Florida |
Recruiting |
| Bioresearch Partner - Hialeah Hospital |
Hialeah |
Florida |
Recruiting |
| D&H National Research Centers - Cancer Research Center, LLC |
Margate |
Florida |
Recruiting |
| Urology Center of Iowa Research |
Clive |
Iowa |
Recruiting |
| The Urology Center, P.C. |
Omaha |
Nebraska |
Completed |
| Rutgers Cancer Institute of New Jersey |
New Brunswick |
New Jersey |
Recruiting |
| University of Rochester Medical Center |
Rochester |
New York |
Not Yet Recruiting |
| Montefiore Medical Center - Bronx |
The Bronx |
New York |
Recruiting |
| Urology Specialists of the Carolinas, PLLC |
Charlotte |
North Carolina |
Recruiting |
| Urology Specialists of the Carolinas - Greensboro |
Greensboro |
North Carolina |
Recruiting |
| University of Cincinnati Medical Center (UCMC) |
Cincinnati |
Ohio |
Recruiting |
| The James Cancer Hospital and Solove Research Institute |
Columbus |
Ohio |
Recruiting |
| Urologic Specialists of Oklahoma |
Tulsa |
Oklahoma |
Recruiting |
| Penn State Milton S Hershey Medical Center |
Hershey |
Pennsylvania |
Recruiting |
| Medical University of South Carolina |
Charleston |
South Carolina |
Recruiting |
| Carolina Urologic Research Center, LLC |
Myrtle Beach |
South Carolina |
Recruiting |
| Low county Urology Clinics |
North Charleston |
South Carolina |
Recruiting |
| Amarillo Urology Research |
Amarillo |
Texas |
Completed |
| UPNT Research Institute, LLC |
Arlington |
Texas |
Recruiting |
| Texas Urology Specialists-Austin |
Austin |
Texas |
Recruiting |
| The University of Texas Southwestern Medical Center |
Dallas |
Texas |
Completed |
| Houston Metro Urology (HMU) - Southwest Location |
Houston |
Texas |
Recruiting |
| Lumi Research - Houston |
Houston |
Texas |
Recruiting |
| USA Operation d/b/a US Clinical Trials |
San Antonio |
Texas |
Recruiting |
| Renovatio Clinical - The Woodlands Research Center |
The Woodlands |
Texas |
Recruiting |
| University of Virginia Comprehensive Cancer Center |
Charlottesville |
Virginia |
Recruiting |
| University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv |
Plovdiv |
Plovdiv |
Recruiting |
| Multiprofile Hospital for Active Treatment - Shumen, Department of Urology |
Shumen |
Shumen |
Recruiting |
| MHAT Haskovo AD |
Haskovo |
Bulgaria |
Recruiting |
| University Multiprofile Hospital for Active Treatment and Emergency Medicine "N.I. Pirogov," Sofia, Clinic of Urology |
Sofia |
Bulgaria |
Recruiting |
| LTD West Regional Center of Modern Medical Technologies |
Kutaisi |
Imereti |
Recruiting |
| Guram Karazanashvili Robotic Center LLC |
Tbilisi |
K'alak'i T'bilisi |
Recruiting |
+ 39 more sites — see the full list on the official registry below.
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