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Safety of Pre-TURBT Intravesical Instillation of Escalating Doses of TC-3 Gel and MMC in NMIBC Patients

Phase 2
Completed
Conditions
Bladder Cancer
Neoplasms
Urologic Diseases
Urinary Bladder Diseases
Interventions
Drug: 120 mg MMC in 90ml gel
Drug: 140 mg MMC in 90ml gel
Drug: 140 mg MMC in 60ml gel
Drug: 160 mg MMC in 90ml gel
Drug: 160 mg MMC in 60ml gel
Drug: 120 mg MMC in 60ml gel
Registration Number
NCT02307487
Lead Sponsor
UroGen Pharma Ltd.
Brief Summary

A prospective, open label, modified 3+3 dose escalation study. This dose-escalation study is designed to carefully assess the safety of successive cohorts of patients (3 patients/cohort), each cohort treated with a fixed dose of TC-3 and MMC Intravesical instillations.

Detailed Description

A prospective, open label, modified 3+3 dose escalation study. This dose-escalation study is designed to carefully assess the safety of successive cohorts of patients. A total of 10 patients will be treated in the first cohort with 120mg MMC-TC-3 Gel unless DLT is reached in more than 1/3 of the patients in the cohort. The next cohorts will have 3 patients/cohort, each cohort treated with a fixed dose of TC-3 and MMC Intravesical instillations. Since data is already available for 40 and 80 mg MMC in TC-3 gel, the initial cohort is 120mg MMC mixed with of 60 mL TC-3. Subsequent cohorts will be given dose levels of 140mg \& 160mg MMC mixed with 60 mL TC-3. Thus, if 160mg MMC mixed in 60ml TC-3 will be found to be safe and tolerable, no higher doses will be further explored at this stage.

If 120 mg MMC in 60 ml TC-gel (2 mg/ml) is found to be intolerable, higher concentrations will not be tested. Instead, a dose of 120 mg in 90 cc TC gel and a subsequent doses of 140 and 160 mg MMC in 90 cc TC gel will be tested in the same dose escalating manner. This will allow testing similar doses at lower concentrations (up to 1.78 mg/ml) but with a longer dwell time due to the larger volume of TC-gel.

Dose escalation is to be halted when the maximum tolerated dose (MTD) will be reached; MTD is defined as one dose level below which dose limited toxicity (DLT) is Any adverse event (AE) related to TC-3+MMC and qualified per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) as grade 3 or 4 will be evaluated by board of 2 independent physicians to determine if it qualifies as DLT based on the known safety profile of MMC. If the NCI grade will not apply, the adverse event to be graded as mild, moderate, or severe.

If one of the three patients in a cohort experienced a DLT, three more patients will be added to the cohort for AE confirmation, only if 3 patients from the given cohort will experienced DLT, the MTD will be reached If no further DLTs are observed in the cohort, the three patients will be enrolled in the next successive cohort.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
14
Inclusion Criteria
  • Patient is 18 years of age or older.
  • Patient has signed Informed Consent Form and is willing and able to abide by the protocol.
  • Patients diagnosed with Low Grade (LG) or High grade (HG) NMIBC.
  • No active urinary tract infection as confirmed by urine culture.
  • If the patient is a female of childbearing potential, she is using two acceptable & effective methods of contraception, until 6 months post treatment
  • A negative serum pregnancy test at screening for female patient with childbearing potential
  • If the patient is a male he should use a condom during intercourse, for at least 48 hours post each instillation.
  • If the patient is a male that has a partner that is a female of childbearing potential, he should be advised to use two acceptable & effective methods of contraception until 6 months post treatment.
Exclusion Criteria
  • Tumor located in prostatic urethra (for male patient) or in the bladder diverticulum.
  • Prior or required pelvic radiotherapy.
  • Systemic chemotherapy within 1 year prior the screening.
  • Pregnant or breastfeeding female patient.
  • Treatment of bladder cancer with BCG within the last 12 months prior to screening visit . Exception: For patient that had BCG treatment within 6-12 months prior to screening visit and are not symptomatic, the patient may enroll at the investigator discretion.
  • Treatment (full course) with intravesical chemotherapy within the 3 months, prior to screening visit.
  • Contraindication to MMC treatment as per investigator determination, or known sensitivity to MMC or TC-3 ingredients.
  • The patient has a known current urinary retention which requires intermittent catheterization to empty the bladder.
  • The patient has a bleeding disorder or a screening platelet count <100X109/L.
  • The patient has screening hemoglobin <10g/dL OR white blood cells < 4000 mm3.
  • GFR<30
  • Hepatic values exceeding 2 times the upper normal limit.
  • The patient has a concurrent severe and/or uncontrolled medical condition (e.g., uncontrolled diabetes, compensated congestive heart failure [NYHA III and over], myocardial infarction within 6 months of screening visit, unstable or uncontrolled hypertension or an active uncontrolled infection) or psychiatric disease, which could compromise participation, compliance with scheduled visits, and/or completion of the study in the opinion of the investigator.
  • The patient has a documented severe vesico-ureteral reflux or has an in-dwelling ureteral stent.
  • The patient participated in an investigational interventional study within the past 90 days, prior to screening visit.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Cohort A2120 mg MMC in 90ml gel120 mg MMC in 90ml gel
Cohort B2140 mg MMC in 90ml gel140 mg MMC in 90ml gel
Cohort B140 mg MMC in 60ml gel140 mg MMC in 60ml gel
Cohort C2160 mg MMC in 90ml gel160 mg MMC in 90ml gel
Cohort C160 mg MMC in 60ml gel160 mg MMC in 60ml gel
Cohort A120 mg MMC in 60ml gel120 mg MMC in 60ml gel
Primary Outcome Measures
NameTimeMethod
Vital signs findings considered to be dose limiting according to the CTCAE V 4.06 weeks
Clinical evaluation findings considered to be dose limiting according to the CTCAE V 4.06 weeks
Rate of Adverse Events (AE) findings considered to be dose limiting according to the CTCAE V 4.06 weeks
Lab results considered to be dose limiting according to the CTCAE V 4.06 weeks
Secondary Outcome Measures
NameTimeMethod
Vital signs and laboratory findings15 months
Rates of all adverse events or clinically relevant physical examination15 months
MMC maximum plasma concentration and concentration time curve during 6 hr post instillation15 months
Duration of MMC retention in the bladder as detected by urine MMC levels post instillation (6-7 hours)15 months

Trial Locations

Locations (2)

Wolfson Medical Center of Holon, Department of Urology

🇮🇱

Holon, Israel

Meir Medical Center

🇮🇱

Kfar Saba, Israel

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