MITOMYCIN for Non-muscle invasive bladder cancer

Inclusion criteria

• {"criterion_text":"- 1. Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol."}
• {"criterion_text":"- 2. Patient must be ≥ 18 years of age at the time of signing the ICF."}
• {"criterion_text":"- 3. Patient who has LG NMIBC (Ta) histologically confirmed by cold cup biopsy at Screening or within 8 weeks before Screening."}
• {"criterion_text":"- 4. History of LG NMIBC requiring treatment with TURBT. Note: This refers to a previous episode(s) and not to the current episode for which the patient is being screened."}
• {"criterion_text":"- 5. Has intermediate risk disease, defined as having 1 or 2 of the following: a. Presence of multiple tumors. b. Solitary tumor > 3 cm. c. Early or frequent recurrence (≥ 1 occurrence of LG NMIBC within 1 year of the current diagnosis at the initial Screening Visit)."}
• {"criterion_text":"- 6. Negative voiding cytology for HG disease within 8 weeks before Screening."}
• {"criterion_text":"- 7. Has adequate organ and bone marrow function as determined by routine laboratory tests as below: • Leukocytes ≥ 3,000/μL (≥ 3 × 109/L). • Absolute neutrophil count ≥ 1,500/μL (≥ 1.5 × 109/L). • Platelets ≥ 100,000/μL (≥ 100 × 109/L). • Hemoglobin ≥ 9.0 g/dL. • Total bilirubin ≤ 1.5 × upper limit of normal (ULN). • Aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) ≤ 2.5 × ULN. • Alkaline phosphatase (ALP) ≤ 2.5 × ULN. • Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min"}
• {"criterion_text":"- 8. Has an anticipated life expectancy of at least the duration of the trial."}
• {"criterion_text":"- 9. Both male and female patients: Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. a.Female partner of male patient: Willing to use 2 acceptable forms of effective contraception from enrollment through 6 months post treatment if the female partner is of childbearing potential (defined as premenopausal women who have not been sterilized). Acceptable methods of birth control which are considered to have a low failure rate (ie, less than 1% per year) when used consistently and correctly, such as 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. b.Female patient: Willing to use 2 acceptable forms of effective contraception from enrollment through 6 months post treatment if the patient is of childbearing potential (defined as premenopausal women who have not been sterilized)."}

Exclusion criteria

• {"criterion_text":"- 1. Received Bacillus Calmette-Guérin (BCG) treatment for urothelial carcinoma (UC) within previous 1 year."}
• {"criterion_text":"- 10. Is pregnant or breastfeeding."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 12. History of prior treatment with an intravesical chemotherapeutic agent in the past 2 years except for a single dose of chemotherapy immediately after any previous TURBT."}
• {"criterion_text":"- 13. Has participated in a study with an investigational agent or device within 30 days of enrollment."}
• {"criterion_text":"- 14. Has previously participated in a study in which they received UGN-102."}
• {"criterion_text":"- 15. Has any other active malignancy requiring treatment with systemic anticancer therapy (eg, chemotherapy, immunotherapy, radiation therapy). Superficial cancers such as cutaneous basal cell or squamous cell carcinomas that can be treated locally are allowed."}
• {"criterion_text":"- 16. Has any other clinically significant medical or surgical condition that in the Investigator’s opinion could compromise patient safety or the interpretation of study results."}
• {"criterion_text":"- 2. History of HG bladder cancer (papillary or carcinoma in situ [CIS]) in the past 2 years."}
• {"criterion_text":"- 3. Known allergy or sensitivity to mitomycin that in the Investigator’s opinion cannot be readily managed."}
• {"criterion_text":"- 4. Clinically significant urethral stricture that would preclude passage of a urethral catheter."}
• {"criterion_text":"- 5. History of: a. Neurogenic bladder. b. Active urinary retention. c. Any other condition that would prohibit normal voiding."}
• {"criterion_text":"- 6. Past or current muscle invasive bladder cancer (ie, T2, T3, T4) or metastatic UC."}
• {"criterion_text":"- 7. Current tumor grading of T1."}
• {"criterion_text":"- 8. Concurrent upper tract urothelial carcinoma (UTUC)."}
• {"criterion_text":"- 9. Evidence of active urinary tract infection (UTI) that in the Investigator’s opinion cannot be treated and resolved prior to biopsy and/or administration of study treatment."}

Primary endpoints

• {"endpoint_text":"- CRR, defined as the proportion of patients who achieved CR at the 3 month Visit (3 months after the first instillation of UGN-102) as determined by cystoscopy, for cause biopsy, and urine cytology.","definition_or_measurement_approach":"Proportion of patients who achieved complete response (CR) at the 3-month visit (3 months after first instillation), determined by cystoscopy, for-cause biopsy and urine cytology."}

Secondary endpoints

• {"endpoint_text":"- 1. DOR in patients who achieved CR at the 3 month Visit, defined as the time from the date of evidence of CR at the 3 month Visit to the earliest date of recurrence or progression as determined using the date of cystoscopy, for cause biopsy, or cytology, or death due to any cause, whichever occurred first.","definition_or_measurement_approach":"Time from date of documented CR at 3-month visit to earliest date of recurrence/progression per cystoscopy, for-cause biopsy, cytology, or death."}
• {"endpoint_text":"- 2. DCR rate at scheduled disease assessment time points, defined as the proportion of patients who achieved CR at the 3-month Visit and maintained CR (ie, no detectable disease) up to that particular follow up disease assessment.","definition_or_measurement_approach":"Proportion of patients with sustained CR (no detectable disease) at scheduled follow-up assessment time points after achieving CR at 3 months."}
• {"endpoint_text":"- 3. DFS in patients who achieved CR at the 3 month Visit, defined as the time from first dose to the earliest date of recurrence or progression as determined using the date of cystoscopy, for cause biopsy, or cytology, or death due to any cause, whichever occurred first.","definition_or_measurement_approach":"Time from first dose to earliest date of recurrence/progression per cystoscopy, for-cause biopsy, cytology, or death."}
• {"endpoint_text":"- 4. The safety profile of UGN-102 will be evaluated through the reporting of AEs, including SAEs and AESIs, and through standard clinical and laboratory tests (eg, hematology and chemistry, urinalysis, physical examination, and vital signs).","definition_or_measurement_approach":"Assessment of adverse events (AEs), serious AEs (SAEs), AEs of special interest (AESIs), and routine clinical/laboratory tests (hematology, chemistry, urinalysis, physical exam, vital signs)."}
• {"endpoint_text":"- Exploratory 1. TTR in patients who were NCR (residual disease) at the 3-month Visit, defined as the time from the date of first treatment after NCR to the earliest date of recurrence or progression as determined using the date of cystoscopy, for cause biopsy, or cytology, whichever occurred first.","definition_or_measurement_approach":"Time from first treatment after NCR to earliest date of recurrence/progression per cystoscopy, for-cause biopsy, or cytology."}
• {"endpoint_text":"- Exploratory 2. Number (%) of response outcomes evaluated at the first disease assessment visit after SOC.","definition_or_measurement_approach":"Counts and percentages of response outcomes at first disease assessment visit following standard of care (SOC) treatment."}
• {"endpoint_text":"- Exploratory 3. Proportion of patients whose planned NMIBC treatment at baseline was downgraded following treatment with UGN-102 (eg, from TURBT to biopsy and/or fulguration).","definition_or_measurement_approach":"Proportion of patients with reduction in planned baseline NMIBC treatment intensity after UGN-102 (e.g., TURBT → biopsy/fulguration)."}
• {"endpoint_text":"- Exploratory 4. Changes from baseline in patient scores on the QLQ C30 and QLQ-NMIBC24 questionnaires.","definition_or_measurement_approach":"Change from baseline in patient-reported outcomes measured by QLQ-C30 and QLQ-NMIBC24 instruments."}

Other endpoints

• {"endpoint_text":"- Exploratory 1. TTR in patients who were NCR (residual disease) at the 3-month Visit, defined as the time from the date of first treatment after NCR to the earliest date of recurrence or progression as determined using the date of cystoscopy, for cause biopsy, or cytology, whichever occurred first.","definition_or_measurement_approach":"See secondary endpoint definition: Time from first treatment after NCR to earliest recurrence/progression per cystoscopy, for-cause biopsy, or cytology."}
• {"endpoint_text":"- Exploratory 2. Number (%) of response outcomes evaluated at the first disease assessment visit after SOC.","definition_or_measurement_approach":"See secondary endpoint definition: counts/percentages of response outcomes at first disease assessment after SOC."}
• {"endpoint_text":"- Exploratory 3. Proportion of patients whose planned NMIBC treatment at baseline was downgraded following treatment with UGN-102 (eg, from TURBT to biopsy and/or fulguration).","definition_or_measurement_approach":"See secondary endpoint definition: proportion with downgraded baseline treatment."}
• {"endpoint_text":"- Exploratory 4. Changes from baseline in patient scores on the QLQ C30 and QLQ-NMIBC24 questionnaires.","definition_or_measurement_approach":"See secondary endpoint definition: change from baseline in QLQ-C30 and QLQ-NMIBC24 scores."}

Austria

Earliest CTIS Part Ii Submission Date

21-10-2024

Latest Decision Or Authorization Date

22-03-2026

Processing Time Days

517

Number Of Sites

1

Number Of Participants

2

Estonia

Earliest CTIS Part Ii Submission Date

21-10-2024

Latest Decision Or Authorization Date

02-04-2026

Processing Time Days

528

Number Of Sites

3

Number Of Participants

14

Latvia

Earliest CTIS Part Ii Submission Date

21-10-2024

Latest Decision Or Authorization Date

15-04-2026

Processing Time Days

541

Number Of Sites

4

Number Of Participants

38

Lithuania

Earliest CTIS Part Ii Submission Date

21-10-2024

Latest Decision Or Authorization Date

08-05-2026

Processing Time Days

564

Number Of Sites

2

Number Of Participants

7

Bulgaria

Earliest CTIS Part Ii Submission Date

21-10-2024

Latest Decision Or Authorization Date

16-04-2026

Processing Time Days

542

Number Of Sites

5

Number Of Participants

85

Spain

Earliest CTIS Part Ii Submission Date

21-10-2024

Latest Decision Or Authorization Date

21-04-2026

Processing Time Days

547

Number Of Sites

4

Number Of Participants

8

Poland

Earliest CTIS Part Ii Submission Date

21-10-2024

Latest Decision Or Authorization Date

20-04-2026

Processing Time Days

546

Number Of Sites

1

Number Of Participants

7

Primary sponsor

Full Name

Urogen Pharma Ltd.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Israel

Third parties

• {"country":"United States","full_name":"Quest Diagnostics Nichols Institute Inc.","duties_or_roles":"[{\"id\":915711,\"code\":\"4\"}]","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"[{\"id\":915700,\"code\":\"3\"}]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"ClinChoice","duties_or_roles":"[{\"id\":915703,\"code\":\"6\"}]","organisation_type":"Industry"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"[{\"id\":915701,\"code\":\"8\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Q2 Solutions LLC","duties_or_roles":"[{\"id\":915704,\"code\":\"4\"}]","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Sweden","full_name":"Viedoc Technologies AB","duties_or_roles":"[{\"id\":915702,\"code\":\"15\",\"value\":\"ePro - Patient Questionnaires\"}]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"[{\"id\":915713,\"code\":\"7\"}]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Germany","full_name":"Catalent Germany Schorndorf GmbH","duties_or_roles":"[{\"id\":915712,\"code\":\"15\",\"value\":\"Ancillary and solvent Supplies\"}]","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Psi Cro AG","duties_or_roles":"[{\"id\":915705,\"code\":\"1\"},{\"id\":915706,\"code\":\"11\"},{\"id\":915707,\"code\":\"12\"},{\"id\":915708,\"code\":\"15\",\"value\":\"Insurance procurement\"},{\"id\":915709,\"code\":\"2\"},{\"id\":915710,\"code\":\"5\"}]","organisation_type":"Pharmaceutical company"}