Low Grade Upper Tract Urothelial Cancer MedDRA version: 21.1 Level: LLT Classification code 10077840 Term: Urothelial cancer of renal pelvis System Organ Class: 100000004864 MedDRA version: 20.0 Level: LLT Classification code 10046375 Term: Ureter cancer System Organ Class: 100000004864
Conditions
Sponsors
Steba Biotech, S.A.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Male and female patients 18 years or older 2.Able to understand and provide written informed consent and willing to comply with all tests and procedures associated with the study 3.New or recurrent low-grade, non-invasive UTUC disease 4.Biopsy-proven disease. A concurrence of the central pathology reader will be required for eligibility. 5.Up to 2 biopsy-proven sites of low-grade involvement with the largest tumor (index tumor) between 5 mm and 15 mm in diameter (as measured by endoscopy), both located in the calyces, renal pelvis or in the ureter of ipsilateral kidney, with an absence of high-grade cells on cytology. (Ureter involvement should be in one anatomical location with no more than 20 mm of contiguous ureteral length). 6.Karnofsky Performance Status = 50% 7.Adequate organ function defined at baseline as: ?ANC =1,000/ µl, ?Platelets =75,000/ µl, Hb =9 g/dl, ?INR =2 ?Estimated glomerular filtration rate eGFR =30 ml/min using CKD-EPI Method ?Total serum bilirubin =65 years) yes F.1.3.1 Number of subjects for this age range 70
Exclusion criteria
Exclusion criteria: 1.Current high-grade or muscle invasive (>pT1) urothelial carcinoma of the bladder 2.Carcinoma in situ (CIS) current or previous in the upper urinary tract 3.History of invasive T2 or higher urothelial cancer in past 2 years 4.Participation in another clinical study involving an investigational product within 1 month before study entry 5.BCG or local chemotherapy treatment in the upper urinary tract within 2 months prior to inclusion 6.Systemic chemotherapy treatment within 2 months prior to enrollment 7.Prohibited medication that could not be adjusted or discontinued prior to study treatment 8.Any other medical or psychiatric co-morbidities, including decompensated heart failure, unstable angina or coronary artery disease, severe pulmonary or liver disease or current heavy smoker that, in the opinion of the study investigator, would make the patient a poor candidate for the study 9.Pregnant or breast-feeding women Women of childbearing potential (WOCBP) must undergo a negative serum pregnancy test prior to study entry. 10.Men and women of reproductive potential not willing to observe conventional and effective birth control for the duration of treatment and for 90 days following the last TOOKAD VTP treatment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the efficacy and durability of effect following TOOKAD (padeliporfin) VTP on low grade UTUC tumors in the calyces, renal pelvis and ureter ;Secondary Objective: -To evaluate TOOKAD (padeliporfin) VTP related safety and tolerability in the treatment of low grade UTUC tumors in the calyces, renal pelvis and ureter -To explore a potential link between UTUC cancer genomic markers and treatment related clinical outcomes and disease progression ;Primary end point(s): The primary efficacy outcome is the absence of UTUC tumors in the entire ipsilateral calyces, renal pelvis and ureter on endoscopic evaluation. This outcome will be determined dichotomously as either failure or success in achieving complete response. Complete Response will be defined as absence of disease based on: •absence of visual tumor on endoscopy •no evidence of tumor on biopsy (if feasible) •negative urinary cytology by instrumented collection ;Timepoint(s) of evaluation of this end point: This endpoint will be evaluated at the time of Primary Response Evaluation (PRE) (28 +/- 3 days post last treatment) during the TOOKAD (padeliporfin) VTP induction treatment phase. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1.The duration of response at the entire ipsilateral kidney will be defined as absence of disease in the entire ipsilateral calyces, renal pelvis and ureter 2.The duration of response at the Treatment Area of the ipsilateral kidney will be defined as absence of disease in the ipsilateral Treatment Area 3.Overall renal functional outcome 4.Kidney organ loss or preservation 5.Pathological evaluation of response performed in kidney tissue of patient that will undergo kidney surgical removal following at least ne TOOKAD VTP treatment 6.Safety follow up based and recording of adverse events;Timepoint(s) of evaluation of this end point: 1.Measured at the 3, 6, 9 and 12 months maintenance treatment visit post PRE 2.Measured at the 3, 6, 9 and 12 months maintenance treatment visit post PRE 3.Measured at the 6, 12, 18 and 24 months maintenance treatment visit post PRE 4.Measured at the 3, 6, 9 and 12, 18 and 24 months maintenance treatment visit post PRE 5.Whenever possible for patients that will undergo kidney surgical removal 6.At 18 and 24 months post PRE or 6 and 12 months post PRE if the patient has not participated to Maintenance treatment phase | — |
Countries
Austria, France, Germany, Israel, Italy, Portugal, Spain, United Kingdom, United States
Contacts
Public ContactHead of Global R&D
STEBA Biotech S.A.
Outcome results
None listed