ENdoluminal LIGHT ActivatED Treatment of Upper Tract Urothelial Cancer (ENLIGHTED) Study

NCT04620239 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: CLIN2001 UCM301
• Study Type: interventional
• Target: 100
• Locations: 5 countries
• Sites: 18

Brief Summary

This is a phase 3, open label, single arm study of padeliporfin in the treatment of Upper Tract Urothelial Carcinoma (UTUC). The ENLIGHTED study will recruit patients with low-grade non-invasive upper tract urothelial carcinoma in either the kidney or the ureter. Patients will be treated with padeliporfin VTP in two phases: an Induction Treatment Phase and a Maintenance Treatment Phase and will be followed up for up to an additional 48 months in the long term (non intervention) follow up phase with the specific duration depending on the patient's response to treatment

Conditions

Transitional Cell Cancer of Renal Pelvis and Ureter

Keywords

UTUC; Low Grade

Outcome Measures

Primary Outcomes (1)

• Number of patients with absence of UTUC tumors in the entire ipsilateral calyces, renal pelvis and ureter

  Primary efficacy outcome is the absence of UTUC tumors in the entire ipsilateral calyces renal pelvis and ureter on endoscopic evaluation at the time of Primary Response Evaluation (PRE) (28 +/- 3 days post last treatment) during padeliporfin VTP induction treatment phase. 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

  28 +/- 3 days post last treatment

Secondary Outcomes (17)

• Duration of response at the entire ipsilateral kidney

  12 months post PRE

• Duration of response at the entire ipsilateral kidney

  3, 6, 9 months post PRE

• Duration of response at the Treatment Area of the ipsilateral kidney

  3, 6, 9, and 12 months post PRE

• Overall renal function

  6 and 12 months post PRE

• Kidney organ loss or preservation

  3, 6, 9, and 12 months post PRE

Study Arms (1)

padeliporfin VTP

EXPERIMENTAL

Induction Treatment phase:1-3 padeliporfin VTP treatments provided 4 weeks (28 +/-3 days) apart. Maintenance Treatment Phase: Repeated maintenance VTP treatments during this period will be provided for patients who show evidence of tumor recurrence that is deemed treatable.

Drug: padeliporfin VTP

Interventions

padeliporfin VTP DRUG

During treatment, placement at the target area of an optical light fiber, through the working channel of the ureteroscope. Intravenous administration of padeliporfin at the dose of 3.66 mg/kg infused over 10 minutes. Each target area will be illuminated for 10 minutes.

Also known as: WST11

padeliporfin VTP

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Steba Biotech S.A.: lead
• PrimeVigilance: collaborator
• ICON plc: collaborator

Study Sites (18)

University of California - Irvine Medical Center

Irvine, California, 92868, United States

RECRUITING

Keck School of Medicine at USC Medical Center

Los Angeles, California, 90033, United States

RECRUITING

Emory University Hospital

Atlanta, Georgia, 30322, United States

RECRUITING

The Johns Hopkins Hospital, The Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States

RECRUITING

Albany Medical College

Albany, New York, 12208, United States

RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

RECRUITING

The Ohio State University (OSU)

Columbus, Ohio, 43210, United States

RECRUITING

The Pennsylvania State University (Penn State) Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

RECRUITING

Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425, United States

RECRUITING

Carolina Urologic Research Center

Myrtle Beach, South Carolina, 29572, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75390, United States

RECRUITING

University of Washington

Seattle, Washington, 98195, United States

RECRUITING

CHU de Lille - Hopital Claude Huriez

Lille, 59037, France

RECRUITING

HCL Hopital Edouard Herriot

Lyon, 69437, France

RECRUITING

Universitaetsklinikum Tuebingen

Tübingen, 72076, Germany

RECRUITING

Sheba Medical Center

Ramat Gan, Israel

RECRUITING

Hospital Universitario de A Coruña

A Coruña, 15006, Spain

RECRUITING

Hospital Universitario Vall d'Hebron

Barcelona, 08035, Spain

RECRUITING

MeSH Terms

Interventions

padeliporfin

Study Officials

• Vitaly Margulis, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Eyal Morag, MD

CONTACT

+97253 9656070; eyal.morag@impactbiotech.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Masking Details: Open label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Phase III, single arm, non randomized, multicenter trial

Study Record Dates

• First Submitted: November 5, 2020
• First Posted: November 6, 2020
• Study Start: March 22, 2021
• Primary Completion: March 30, 2026
• Study Completion (Estimated): January 25, 2029
• Last Updated: February 4, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (12); IL (1); ES (2); FR (2); DE (1)