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

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