Multicenter Phase 3 Pivotal Study to Evaluate the Safety and Efficacy of TOOKAD (padeliporfin) Vascular Targeted Photodynamic Therapy in the Treatment of Low-Grade Upper Tract Urothelial Cancer
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 14
- 主要终点
- 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.
研究概览
简要总结
To demonstrate the efficacy and durability of effect following TOOKAD (padeliporfin) VTP on low grade UTUC tumors in the calyces, renal pelvis and ureter
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Long Term Follow-up Phase
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Male and female patients 18 years or older
- •Able to understand and provide written informed consent and willing to comply with all tests and procedures associated with the study
- •New or recurrent low-grade, non-invasive UTUC disease
- •Biopsy-proven disease. A concurrence of the central pathology reader will be required for eligibility
- •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 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)
- •Karnofsky Performance Status ≥ 50%
- •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 <3 mg/dL, AST/ALT ≤5× upper limit of normal
排除标准
- •Current high-grade or muscle invasive (>pT1) urothelial carcinoma of the bladder
- •Pregnant or breast-feeding women; Women of childbearing potential (WOCBP) must undergo a negative serum pregnancy test prior to study entry
- •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
- •Carcinoma in situ (CIS) current or previous in the upper urinary tract
- •History of invasive T2 or higher urothelial cancer in past 2 years
- •Participation in another clinical study involving an investigational product within 1 month before study entry
- •BCG or local chemotherapy treatment (including VEGF-targeted therapy) in the upper urinary tract within 2 months prior to inclusion
- •Systemic chemotherapy treatment (including VEGF-targeted therapy) within 2 months prior to enrollment
- •Prohibited medication that could not be adjusted or discontinued prior to study treatment
- •Patients with photosensitive skin diseases or porphyria
- •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
结局指标
主要结局
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.
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
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
Additional information are part of the protocol (section Primary Endpoint/Estimand)
Additional information are part of the protocol (section Primary Endpoint/Estimand)
次要结局
- 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
- The duration of response at the Treatment Area of the ipsilateral kidney will be defined as absence of disease in the ipsilateral Treatment Area
- Overall renal functional outcome
- Kidney organ loss or preservation
- Pathological evaluation of response performed in kidney tissue of patient that will undergo kidney surgical removal following at least one TOOKAD (padeliporfin) VTP treatment
- Safety follow up based and recording of adverse events
- Additional information are part of the protocol (section Secondary Endpoint/Estimand)
研究者
Global Head RA&PV
Scientific
Steba Biotech S.A.