Treatment of low-grade upper tract urothelial cancer with padeliporfin
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
This study is testing if a new treatment called padeliporfin can help people with low-grade non-invasive upper tract urothelial cancer in the kidney or ureter feel better and stay cancer-free.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Steba Biotech S.A. Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 18 sites (Irvine, California and 17 other locations) |
| Trial ID | NCT04620239 on ClinicalTrials.gov |
What this trial studies
This phase 3, open-label, single-arm study evaluates the efficacy of padeliporfin in treating patients with low-grade non-invasive upper tract urothelial carcinoma (UTUC) affecting the kidney or ureter. Participants will undergo an induction treatment phase consisting of 1-3 padeliporfin vascular-targeted photodynamic therapy (VTP) treatments, followed by a maintenance phase for those who achieve complete response. The study aims to assess the treatment's success through endoscopic evaluations and urinary cytology over a follow-up period of up to 48 months.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with biopsy-proven low-grade, non-invasive UTUC and specific tumor size criteria.
Not a fit: Patients with high-grade UTUC or those requiring extirpative surgery for their condition may not benefit from this treatment.
Why it matters
Potential benefit: If successful, this treatment could provide a non-invasive option for patients with low-grade UTUC, potentially preserving kidney function and avoiding more radical surgical interventions.
How similar studies have performed: Other studies have shown promise with vascular-targeted photodynamic therapies, suggesting potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 in the ureter of the 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 giltration rate (eGFR) ≥30 ml/min (using CKD-EPI Method) * Total serum bilirubin \<3 mg/dL, AST/ALT ≤5× upper limit of normal Exclusion Criteria: * Current high-grade or muscle invasive (\>pT1) urothelial carcinoma of the bladder * 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 or 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 * 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 padeliporfin VTP treatment.
Where this trial is running
Irvine, California and 17 other locations
- University of California - Irvine Medical Center — Irvine, California, United States (Recruiting)
- Keck School of Medicine at USC Medical Center — Los Angeles, California, United States (Recruiting)
- Emory University Hospital — Atlanta, Georgia, United States (Recruiting)
- The Johns Hopkins Hospital, The Sidney Kimmel Cancer Center — Baltimore, Maryland, United States (Recruiting)
- Albany Medical College — Albany, New York, United States (Recruiting)
- Memorial Sloan Kettering Cancer Center — New York, New York, United States (Recruiting)
- The Ohio State University (OSU) — Columbus, Ohio, United States (Recruiting)
- The Pennsylvania State University (Penn State) Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States (Recruiting)
- Medical University of South Carolina (MUSC) — Charleston, South Carolina, United States (Recruiting)
- Carolina Urologic Research Center — Myrtle Beach, South Carolina, United States (Recruiting)
- University of Texas Southwestern — Dallas, Texas, United States (Recruiting)
- University of Washington — Seattle, Washington, United States (Recruiting)
- CHU de Lille - Hopital Claude Huriez — Lille, France (Recruiting)
- HCL Hopital Edouard Herriot — Lyon, France (Recruiting)
- Universitaetsklinikum Tuebingen — Tübingen, Germany (Recruiting)
- Sheba Medical Center — Ramat Gan, Israel (Recruiting)
- Hospital Universitario de A Coruña — A Coruña, Spain (Recruiting)
- Hospital Universitario Vall d'Hebron — Barcelona, Spain (Recruiting)
Study contacts
- Principal investigator: Vitaly Margulis, MD — University of Texas Southwestern Medical Center
- Study coordinator: Eyal Morag, MD
- Email: eyal.morag@impactbiotech.com
- Phone: +97253 9656070
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.