A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorUroGen Pharma Ltd.
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
Provide written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and protocol.
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.
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.
Patient should have at least 1 remaining papillary LG tumor evaluated visually with a diameter of at least 5 mm post-biopsy.
Disqualifiers
Patient received Bacillus Calmette-Guérin (BCG) treatment for UC during the 6 months before enrollment.
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.
Patient has a history of carcinoma in situ (CIS) in the urinary tract.
Patient has a history of invasive UC in the past 5 years.
Trial design
Design model
Single group
Treatments tested in this trial
UGN-104
DrugUGN-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.
Treatment groups
70 Participants
are divided into 1 treatment groupGroup A: UGN-104Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Complete response rate (CRR)
CRR is defined as the proportion of patients who achieved CR at the PDE Visit.
Time frame
3 monthsSecondary outcomes
1
Duration of response (DOR)
DOR is defined as the time from the date of evidence of CR at the PDE Visit to the earliest date of recurrence, progression, or death due to any cause, whichever occurred first.
Time frame
12 months2
Durable complete response (DCR) rate
DCR rate at scheduled disease assessment time points, defined as the proportion of patients who achieved CR at the PDE Visit and maintained CR (ie, no detectable disease) up to that particular follow-up disease assessment.
Time frame
12 months3
Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, TEAEs of special interest, and abnormal clinical laboratory test results (hematology and serum chemistry)
The number of patients with each type of event will be summarized.
Time frame
15 months4
Mitomycin plasma concentrations
Mitomycin plasma concentrations will be assessed in a subset of patients treated with UGN-104.
Time frame
0 (pre-instillation), 0.5, 1, 2, 3, 4, 5, and 6 hours after the first instillation of UGN-104Sponsors and contacts
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Source ClinicalTrials.gov