Brief Title
A Phase 2b Study of UGN-102 for Low Grade Intermediate Risk Non-Muscle-Invasive Bladder Cancer
Official Title
A Phase 2b, Single-Arm, Multicenter Trial to Evaluate the Efficacy and Safety of UGN-102 as Primary Chemoablative Therapy in Patients With Low Grade (LG) Non-Muscle-Invasive Bladder Cancer (NMIBC) at Intermediate Risk of Recurrence
Brief Summary
This Phase 2b, single-arm, multicenter study evaluated the efficacy and safety of UGN-102 as
primary chemoablative therapy in patients with low grade intermediate risk
non-muscle-invasive bladder cancer (LG IR NMIBC).
Detailed Description
Eligible patients were treated with 6 once-weekly intravesical instillations of UGN-102.
The ablative effect of UGN-102 was evaluated at the 3-month Visit, which occurred 5 weeks ± 1
week after the last weekly instillation (3 months after initiation of study drug). Response
was determined based on visual evaluation by cystoscopy (appearance, number, and size of the
lesions) and, if there were remaining lesions, by histopathology of the remaining lesions.
Complete response (CR) was defined as having no detectable disease (NDD) and was assessed
visually during cystoscopy and also by urine cytology. In the event that the investigator was
not sure, and there was suspect tissue, a small biopsy was taken from the suspect tissue to
confirm CR in addition to cystoscopy and urine cytology.
Patients who achieved a CR continued to have monthly telephone contacts to document any
adverse events (AEs) and changes in concomitant medications and were assessed at 6, 9, and 12
months after the first instillation of UGN-102 for evidence of disease recurrence. Patients
who had a non-complete response (non-CR) discontinued the study and continued with standard
of care according to their treating physician.
Safety was determined based on a review of AEs, laboratory assessments, and physical
examination findings.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Complete Response (CR) Rate for UGN-102 Treatment
Secondary Outcome
Durable Complete Response (DCR) Rate
Condition
Bladder Cancer
Intervention
UGN-102
Study Arms / Comparison Groups
UGN-102
Description: Patients were treated with 6 once-weekly intravesical instillations of UGN-102.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
63
Start Date
October 15, 2018
Completion Date
October 21, 2020
Primary Completion Date
January 3, 2020
Eligibility Criteria
Inclusion Criteria:
1. Willing and able to sign an informed consent and comply with the protocol.
2. Has newly diagnosed or historic LG NMIBC (Ta) histologically confirmed by cold cup
biopsy at screening or within 6 weeks of screening.
3. Is at intermediate risk of recurrence, 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. Has negative voiding cytology for high grade (HG) disease at or within 6 weeks of
enrollment.
5. Willing to use 2 acceptable forms of effective contraception from enrollment through 6
months post treatment if the participant is female or the female partner of a male
participant and is of childbearing potential (defined as premenopausal women who have
not been sterilized).
6. Has adequate organ and bone marrow function as determined by routine laboratory tests
as below:
- Leukocytes ≥ 3,000 per μL;
- Absolute neutrophil count ≥ 1,500 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 ≤ 2.5 × ULN;
- Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min.
7. Has no evidence of active urinary tract infection (UTI) at Screening and Baseline
visits.
- In the case of symptomatic UTI, the patient will be treated with a full course of
antibiotics, and study drug will be postponed until resolution. In the case of
asymptomatic bacteriuria, the use of prophylactic antibiotics and postponement of
study drug is left to the discretion of the Principal Investigator (PI).
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 2 years.
3. History of HG papillary UC in the past 2 years.
4. Known allergy or sensitivity to mitomycin.
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 urothelial carcinoma (UTUC).
9. Has participated in a study with an investigational agent or device within 30 days of
enrollment.
10. History of prior treatment with an intravesical chemotherapeutic agent with the
exception of a single dose of chemotherapy immediately after any previous
transurethral resection of bladder tumors (TURBT).
11. 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.
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Elyse Seltzer, MD, ,
Location Countries
Israel
Location Countries
Israel
Administrative Informations
NCT ID
NCT03558503
Organization ID
TC-BC-12
Responsible Party
Sponsor
Study Sponsor
UroGen Pharma Ltd.
Study Sponsor
Elyse Seltzer, MD, Study Director, UroGen Pharma
Verification Date
September 2022