PADELIPORFIN for Upper tract urothelial carcinoma (low-grade) | Urothelial carcinoma of renal pelvis | Ureter cancer

Inclusion criteria

• {"criterion_text":"-Male and female patients 18 years or older\n-Able to understand and provide written informed consent and willing to comply with all tests and procedures associated with the study\n-New or recurrent low-grade, non-invasive UTUC disease\n-Biopsy-proven disease. A concurrence of the central pathology reader will be required for eligibility\n-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)\n-Karnofsky Performance Status ≥ 50%\n-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"}

Exclusion criteria

• {"criterion_text":"-Current high-grade or muscle invasive (>pT1) urothelial carcinoma of the bladder\n-Pregnant or breast-feeding women; Women of childbearing potential (WOCBP) must undergo a negative serum pregnancy test prior to study entry\n-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\n-Carcinoma in situ (CIS) current or previous in the upper urinary tract\n-History of invasive T2 or higher urothelial cancer in past 2 years\n-Participation in another clinical study involving an investigational product within 1 month before study entry\n-BCG or local chemotherapy treatment (including VEGF-targeted therapy) in the upper urinary tract within 2 months prior to inclusion\n-Systemic chemotherapy treatment (including VEGF-targeted therapy) within 2 months prior to enrollment\n-Prohibited medication that could not be adjusted or discontinued prior to study treatment\n-Patients with photosensitive skin diseases or porphyria\n-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"}

Primary endpoints

• {"endpoint_text":"-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.","definition_or_measurement_approach":"Determined by endoscopic evaluation and recorded dichotomously as failure or success in achieving complete response."}
• {"endpoint_text":"-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","definition_or_measurement_approach":"Composite definition: absence of visible tumor on endoscopy, no evidence of tumor on biopsy (if feasible), and negative urinary cytology by instrumented collection."}
• {"endpoint_text":"-Additional information are part of the protocol (section Primary Endpoint/Estimand)","definition_or_measurement_approach":"Additional details referenced to protocol section Primary Endpoint/Estimand."}

Secondary endpoints

• {"endpoint_text":"-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","definition_or_measurement_approach":"Defined as continued absence of disease in the entire ipsilateral calyces, renal pelvis and ureter over time."}
• {"endpoint_text":"-The duration of response at the Treatment Area of the ipsilateral kidney will be defined as absence of disease in the ipsilateral Treatment Area","definition_or_measurement_approach":"Defined as continued absence of disease in the ipsilateral Treatment Area over time."}
• {"endpoint_text":"-Overall renal functional outcome","definition_or_measurement_approach":"Not further specified in CTIS entry; refers to assessment of renal function outcomes."}
• {"endpoint_text":"-Kidney organ loss or preservation","definition_or_measurement_approach":"Assessment of whether the kidney or part of it was preserved or lost (organ-sparing outcome)."}
• {"endpoint_text":"-Pathological evaluation of response performed in kidney tissue of patient that will undergo kidney surgical removal following at least one TOOKAD (padeliporfin) VTP treatment","definition_or_measurement_approach":"Pathological assessment of resected kidney tissue in patients who undergo surgical removal after at least one VTP treatment."}
• {"endpoint_text":"-Safety follow up based and recording of adverse events","definition_or_measurement_approach":"Safety follow-up with recording and monitoring of adverse events."}
• {"endpoint_text":"-Additional information are part of the protocol (section Secondary Endpoint/Estimand)","definition_or_measurement_approach":"Additional secondary endpoint details referenced to protocol section Secondary Endpoint/Estimand."}

Methods

• Country-specific recruitment materials including Advocacy Factsheet, Advocacy Flyer, Web Posting, Dear Colleague Letter, Participant Brochure and Website materials (documents present for IT, AT, FR, DE, ES).
• Local site-based recruitment through participating hospitals/urology departments listed per country (site contact details provided).

Italy

Earliest CTIS Part Ii Submission Date

22-04-2024

Latest Decision Or Authorization Date

22-11-2024

Processing Time Days

214

Number Of Sites

3

Number Of Participants

10

Austria

Earliest CTIS Part Ii Submission Date

22-04-2024

Latest Decision Or Authorization Date

28-08-2025

Processing Time Days

493

Number Of Sites

1

Number Of Participants

10

France

Earliest CTIS Part Ii Submission Date

22-04-2024

Latest Decision Or Authorization Date

27-02-2026

Processing Time Days

676

Number Of Sites

4

Number Of Participants

10

Germany

Earliest CTIS Part Ii Submission Date

22-04-2024

Latest Decision Or Authorization Date

27-02-2026

Processing Time Days

676

Number Of Sites

2

Number Of Participants

10

Spain

Earliest CTIS Part Ii Submission Date

22-04-2024

Latest Decision Or Authorization Date

18-03-2026

Processing Time Days

695

Number Of Sites

4

Number Of Participants

10

Primary sponsor

Full Name

Steba Biotech S.A.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Luxembourg

Contract research organisations

Name

Medpace Reference Laboratories LLC

Responsibilities

Sample centralization and (PK and Genomics) sample storage

Name

Primevigilance Limited

Responsibilities

SUSAR reporting (pharmacovigilance)

Name

MEDPACE LABORATORIES

Responsibilities

Sample centralization and (PK and Genomics) sample storage

Name

Eurofins Adme Bioanalyses

Responsibilities

PK analysis

Name

SocraTec R&D Concepts in Drug Research and Development GmbH

Responsibilities

Study-related duties (code 12 in sponsor duties)

Third parties

• {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"Sample centralization and (PK and Genomics) sample storage","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"Primevigilance Limited","duties_or_roles":"SUSAR reporting","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"MEDPACE LABORATORIES","duties_or_roles":"Sample centralization and (PK and Genomics) sample storage","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Geneuity","duties_or_roles":"Medical image analysis/ review - X-ray, MRI, ultrasound, etc","organisation_type":"Health care"}
• {"country":"France","full_name":"Eurofins Adme Bioanalyses","duties_or_roles":"PK analysis","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"SocraTec R&D Concepts in Drug Research and Development GmbH","duties_or_roles":"Role code 12 (duties listed in record)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"University of Tennessee Medical Center","duties_or_roles":"Central reading for biopsy and pathology","organisation_type":"Educational Institution"}
• {"country":"United States","full_name":"Memorial Sloan Kettering Cancer Center","duties_or_roles":"Genomic","organisation_type":"Health care"}