A Phase III, 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)
Study Description
This Phase 3, single-arm, multicenter study will evaluate the efficacy and safety of UGN-104,
a novel formulation of UGN-101, instilled in the upper urinary tract (UUT) of patients with
LG-UTUC.
Eligibility
Inclusion Criteria
- Provide written informed consent, which includes compliance with the requirements and
restrictions listed in the ICF and in this protocol.
- Patient must be ≥ 18 years of age at the time of informed consent.
- Naive or recurrent patients with LG, non-invasive UTUC in the pyelocalyceal system, with
the following disease characteristics:
a. 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.
b. 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.
c. Patient should have at least 1 remaining papillary LG tumor evaluated visually with a
diameter of at least 5 mm post-biopsy.
d. Wash urine cytology sampled from the pyelocalyceal system documenting the absence of
high-grade (HG) UC, diagnosed not more than 8 weeks before Screening.
e. 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.
- Patients with Eastern Cooperative Oncology Group (ECOG) performance status < 3
(with Karnofsky > 40) (see Appendix 1).
- Patients with life expectancy > 24 months at time of Screening.
- Patients must have adequate organ and bone marrow function as determined by the following
routine laboratory tests:
a. Leukocytes ≥ 3,000/μL (≥ 3 × 109
/L).
b. Absolute neutrophil count ≥ 1,500/μL (≥ 1.5 × 109
/L).
c. Platelets ≥ 100,000/μL (≥ 100 × 109
/L).
d. Hemoglobin ≥ 9.0 g/dL.
e. Total bilirubin ≤ 1.5 × upper limit of normal (ULN)
