This phase II trial studies how well Pemigatinib (an orally administered inhibitor of fibroblast growth factor receptors 1, 2, and 3) works in non-muscle invasive bladder cancer (NMIBC) patients with recurrent tumors and a prior history of low- or intermediate-risk NMIBC tumors. Participants will receive pemigatinib for 4-6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT).
It is a single-arm phase 2 window of opportunity study to assess the antineoplastic activity of pemigatinib in non-muscle invasive bladder cancer (NMIBC) patients with recurrent tumors and a prior history of low- or intermediate-risk NMIBC tumors. Enrolled patients will receive pemigatinib for 4-6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT). The primary endpoint will be complete response rate as determined at TURBT. Secondary endpoints will include safety profile, associations between complete response rate and tumor mutation/fusion status (e.g. FGFR3 specifically, others will also be examined) and NMIBC risk group (low- vs. intermediate-risk), and assessment of pemigatinib post-treatment urothelial tissue concentrations. The study will be conducted at 4-5 high volume bladder cancer institutions within the US and managed through the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center clinical research office.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Participants will take Pemigatinib once daily on days 1 through 28 of each cycle prior to standard of care TURBT.
Sibley Memorial Hospital
Washington D.C., District of Columbia, United States
Johns Hopkins School of Medicine - Sidney Kimmel Comprehensive Cancer Center
Baltimore, Maryland, United States
Associated Medical Professionals Urology
Syracuse, New York, United States
Midlantic Urology
Bala-Cynwyd, Pennsylvania, United States
Keystone Urology
Lancaster, Pennsylvania, United States
Carolina Urologic Research Center
Myrtle Beach, South Carolina, United States
Complete response rate of pemigatinib therapy
The number of participants with a complete response to pemigatinib therapy. Tumor complete response is defined as the complete absence of any stage bladder tumor on post-treatment Transurethral Resection of a Bladder Tumor (TURBT) and no evidence of recurrent urothelial carcinoma on post-treatment urine cytology.
Time frame: 6 weeks
Characterize the safety profile of pemigatinib therapy
Number of Common Terminology Criteria for Adverse Events (CTCAE) grade ≥ 3 toxicities
Time frame: 4 years
Number of Participants with Complete Response and FGFR3 Mutational Status
Time frame: Up to 4 weeks
Number of Participants with Non-Muscle Invasive Bladder Cancer (NMIBC) and Complete Response
Number of participants with complete response rate and baseline NMIBC risk group (low- vs. intermediate-risk)
Time frame: Up to 4 years
Maximal concentration (Cmax, nmol/L) of pemigatinib in urothelial tissue at post-treatment TURBT
Time frame: Up to 4 weeks
Relapse Free Survival (RFS) at 6 months
Number of months from achieving a complete response at initial post-treatment TURBT until relapse.
Time frame: 6 months
Relapse Free Survival (RFS) at 12 months
Number of months from achieving a complete response at initial post-treatment TURBT until relapse.
Time frame: 12 months
Relapse Free Survival (RFS) at 24 months
Number of months from achieving a complete response at initial post-treatment TURBT until relapse.
Time frame: 24 months
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