Clinical Trial Details

• Participants ≥ 18 years of age at the time of informed consent and willing and able to comply with all required study procedures

• Confirmed LG UTUC (both favorable and unfavorable) per AUA

• At least 5mm of marker lesion(s) left behind

• Identification of marker lesion(s) within 8 weeks prior to randomization (refer to Inclusion Criterion #2)

• If synchronous NMIBC, NMIBC must be fully resected and low-grade Ta or T1

• No prior BCG administration within 1 year of date of consent.

• No intravesical chemotherapy within 8 weeks prior to C1D1 (including UGN-101).

• No systemic chemotherapy within 3 months prior to C1D1

• ECOG PS 0-2

• Pathology consists of pure urothelial carcinoma

• Adequate bone marrow function:

  i. Absolute neutrophil count (ANC) ≥1,500/mm3 ii. Platelet count ≥75,000/mm3 iii. Hemoglobin ≥10.0 g/dL

• Adequate liver function:

  i. Total bilirubin ≤ ULN ii. Alanine aminotransferase (ALT) ≤ ULN iii. Aspartate aminotransferase (AST) ≤ ULN

• Adequate renal function:

  i. Estimated glomerular filtration rate >30 mL/min ii. Serum Phosphate level ≤ ULN prior to starting treatment iii. International normalized ratio (INR) ≤1.5 × ULN

Exclusion Criteria:

• Evidence or any features of high grade (HG) UTUC
• History of carcinoma in situ (CIS)
• History of prostatic urethral involvement
• Current or previous history of muscle invasive bladder cancer
• Current or previous history of lymph node positive and/or metastatic bladder cancer
• Evidence of squamous cell carcinoma, adenocarcinoma or undifferentiated carcinoma or small cell of the bladder
• Currently receiving systemic cancer therapy (cytotoxic or immunotherapy)
• Current or prior history of pelvic external beam radiotherapy for bladder cancer
• Current or history of receiving a prior FGFR inhibitor
• Systemic immunotherapy within 6 months prior to C1D1
• Treatment with an investigational agent within 30 days or 5 half-lives from C1D1, whichever is shorter; compounds with an unknown half-life will be default to 30 days.
• Prior treatment with an intravesical or intracavitary agent within 8 weeks of C1D1.
• Current evidence of central serous retinopathy or retinal pigmented epithelial detachment of any grade at time of baseline examination.
• Requiring use of medications that are strong and moderate inhibitors or inducers of CYP3A or inhibitors of P-gp and BCRP transporters (prohibited list of medications)