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

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

INTRODUCTION

  • Org Study ID: CLIN2001 UCM301
  • Secondary ID: N/A
  • NTC ID: NCT04620239
  • Sponsor: Steba Biotech S.A.

BRIEF SUMMARY


This is a phase 3, open label, single arm study of TOOKAD in the treatment of Upper Tract
Urothelial Carcinoma (UTUC). The ENLIGHTED study will recruit patients with low-grade upper
tract urothelial carcinoma in either the kidney or the ureter. The patients will be treated
with TOOKAD (padeliporfin) VTP in two phases: an Induction Treatment Phase and a Maintenance
Treatment Phase and will be followed up for additional 12 months in the long term (non
intervention) follow up phase.

DETAILED DESCRIPTION


Patients entered in the study will undergo an induction treatment phase consisting of 1-3
TOOKAD VTP treatments provided 4 weeks (28 +/-3 days) apart. The goal of this induction
treatment phase will be to achieve Complete Response (CR) in the involved ipsilateral tract
system. During this phase, patients will be treated with TOOKAD (padeliporfin) VTP to
visually identified tumor sites in the calyces,in the renal pelvis and/or ureter and
subsequently examined endoscopically at 28 +/- 3 days post treatment to determine whether the
treatment was successful. If CR was not achieved, additional two treatments of TOOKAD
(padeliporfin) VTP are permitted 28 +/- 3 days apart for a total of up to 3 treatments during
the induction treatment phase. The Primary Response Evaluation (PRE) will be performed 28 +/-
3 days after the last VTP treatment, to determine if the treatment was successful at
achieving CR defined as: absence of visible tumor on endoscopy, negative urinary cytology by
instrumented collection, and no evidence of tumor on biopsy (if feasible). If CR was not
achieved after 3 treatments with TOOKAD (padeliporfin) VTP the treatment will be considered
unsuccessful and the patient will be discontinued from the treatment phases. Patients
achieving CR at the induction treatment phase will be allowed into the maintenance treatment
phase of the study. The patients will then be followed over a period of 12 months post PRE,
to assess the duration of response and its safety, and to provide planned maintenance
treatment.

Repeated maintenance VTP treatments during this period will be provided for patients who show
evidence of tumor recurrence that is deemed treatable as defined by these criteria: low-grade
tumors with the largest tumor (index tumor) betwen 5 mm and 15 mm in diameter, in up to 2
anatomical locations in the calyces, renal pelvis or the ureter (ureter involvement should be
in one anatomical location with no more than 20 mm of contiguous ureteral length). Patients
with treatable tumor recurrence post Induction Treatment Phase would be considered as no
longer having 'complete response in the entire ipsilateral kidney' and time to recurrence
will be recorded.

Patients completing the 12 months of the maintenance treatment phase of the study, or
patients discontinued from the treatment phases after at least one VTP treatment will be
followed for an additional 12 months non intervention to monitor for disease related outcomes
and VTP treatment related adverse events.


  • Overall Status
    Recruiting
  • Start Date
    March 22, 2021
  • Phase
    Phase 3
  • Study Type
    Interventional

PRIMARY OUTCOMES

Primary Outcome 1 - Measure: Number of patients with absence of UTUC tumors in the entire ipsilateral calyces, renal pelvis and ureter

Primary Outcome 1 - Timeframe: 28 +/- 3 days post last treatment

CONDITION

  • Transitional Cell Cancer of Renal Pelvis and Ureter

ELIGIBILITY

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 (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 in the upper urinary tract within 2 months prior
to inclusion

- Systemic chemotherapy treatment within 2 months prior to enrollment

- Prohibited medication that could not be adjusted or discontinued prior to study
treatment

- 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 TOOKAD VTP treatment.

Gender: All

Minimum Age: N/A

Maximum Age: 18 Years

Healthy Volunteers: No

OFFICIAL INFORMATION

Name: Jonathan Coleman, Professor

Role: Principal Investigator

Affiliation: Memorial Sloan Kettering Cancer Center

Overall Contact

Name: Jonathan Coleman, Professor

Phone: (646) 422-4432

Email: colemanj@mskcc.org

LOCATION

Facility Status Contact
Facility: Carolina Urologic Research Center
Myrtle Beach, South Carolina 29572
United States
Status: Recruiting Contact:
Neal Shore, MD
843-449-1010
nshore@gsuro.com