A Phase 3 Study of UGN-102 for Low Grade Intermediate Risk Non-Muscle-Invasive Bladder Cancer
A Randomized, Controlled, Open-Label Study of the Efficacy, Durability, and Safety of UGN-102 With or Without TURBT in Patients With Low Grade Intermediate Risk Non-Muscle-Invasive Bladder Cancer (LG IR NMIBC)
This is a global, randomized, controlled, open-label Phase 3 study designed to assess the long-term efficacy and safety of UGN-102 (mitomycin) for intravesical solution with or without (±) transurethral resection of bladder tumors (TURBT) versus TURBT alone for the treatment of patients with low grade intermediate risk non-muscle-invasive bladder cancer (LG IR NMIBC).
Eligible patients will be randomized in a 1:1 ratio to UGN-102 ± TURBT or TURBT alone. Randomization will be stratified by the presence of a previous LG NMIBC episode within 1 year of the current diagnosis (yes or no). Starting at Day 1, patients randomized to the UGN-102 ± TURBT group will receive 6 once-weekly intravesical instillations of UGN-102 and patients randomized to the TURBT alone group will undergo TURBT. Patients in both treatment groups will return to the clinic at approximately 3 months after the initiation of treatment (7 weeks ± 1 week after the last weekly instillation for the UGN-102 ± TURBT group and 12 weeks ± 1 week after TURBT for the TURBT alone group) to determine response to treatment. Patients determined to have a complete response (CR) will receive no further treatment and will enter the follow-up period of the study. Patients determined to have a non-complete response (NCR) will undergo TURBT of any remaining lesions and will then enter the follow-up period of the study. During the follow-up period, patients will return to the clinic every 3 months to determine durability of response. Patients will remain on study until completion of all follow-up visits (approximately 24 months after the initiation of treatment) or until recurrence or death is documented, whichever occurs first. Patients determined to have a protocol-defined recurrence at any follow-up or unscheduled visit will be considered to have completed the study and released to the care of their treating physician. The study is event-driven and patients may be followed beyond 24 months or additional patients may be enrolled to achieve the target number of events required for the study.
Disease-free survival (DFS)
Time to recurrence (TTR)
Study Arms / Comparison Groups
UGN-102 ± TURBT
Description: 6 once-weekly intravesical instillations of 75 mg UGN-102 starting at Day 1 + TURBT for patients who have a NCR at the 3-month disease assessment (7 weeks ± 1 week after the last weekly instillation of UGN-102).
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
February 19, 2021
Primary Completion Date
Inclusion Criteria: 1. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. 2. Patient who has newly diagnosed or historic LG NMIBC (Ta) histologically confirmed by cold cup biopsy at screening or within 8 weeks of screening. 3. Is at intermediate risk for progression, defined as having 1 or 2 of the following: - Presence of multiple tumors; - Solitary tumor > 3 cm; - Recurrence (≥ 1 occurrence of LG NMIBC within 1 year of the current diagnosis). 4. Negative voiding cytology for high grade (HG) disease within 6 weeks of screening. 5. Has adequate organ and bone marrow function as determined by the following routine laboratory tests: - Leukocytes ≥ 3,000 cells per μL; - Absolute neutrophil count ≥ 1,500 cells per μL; - Platelets ≥ 100,000 per μL; - Hemoglobin ≥ 9.0 g/dL; - Total bilirubin ≤ 1.5 x upper limit of normal (ULN); - Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 × ULN; - Alkaline phosphatase (ALP) ≤ 2.5 × ULN; - Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min. 6. Has no evidence of active urinary tract infection (UTI). 7. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for clinical study participants. Women of childbearing potential (defined as premenopausal women who have not been sterilized), including female patients and female partners of male patients, must be willing to use 2 acceptable forms of effective contraception from enrollment through 6 months post-treatment. Exclusion Criteria: 1. History of carcinoma in situ (CIS) on preliminary cystoscopy within 5 years of enrollment. 2. Received Bacillus Calmette-Guérin (BCG) treatment for urothelial carcinoma (UC) within previous 1 year. 3. History of HG papillary UC in the past 2 years. 4. Known allergy or sensitivity to mitomycin that in the investigator's opinion cannot be readily managed. 5. Clinically significant urethral stricture that would preclude passage of a urethral catheter. 6. History of pelvic radiotherapy. 7. History of: - Neurogenic bladder; - Active urinary retention; - Any other condition that would prohibit normal voiding. 8. Past or current muscle invasive (ie, T2, T3, T4) or metastatic UC or concurrent upper tract UC. 9. Current tumor grading of T1. 10. Has an underlying substance abuse or psychiatric disorder such that, in the opinion of the investigator, the patient would be unable to comply with the protocol. 11. History of prior treatment with an intravesical chemotherapeutic agent except for a single dose of chemotherapy immediately post any previous TURBT. 12. Has previously participated in a study in which they received UGN-102. 13. Has participated in a study with an investigational agent or device within 30 days of randomization.
18 Years - N/A
Accepts Healthy Volunteers
Sandip Prasad, MD, ,
UroGen Pharma Ltd.
Sandip Prasad, MD, Principal Investigator, Atlantic Health System