Materials and Methods: This study is a prospective, single-arm, open-label phase 2 trial for patients with BCG-naïve high-risk NMIBC. Intravesical GemDoce was given weekly for 6 weeks as induction followed by monthly maintenance therapy for 2 years among responders. The primary end point was 3-month complete response, and key secondary end points included adverse events (AEs) and 12-month recurrence-free survival.
Results: Twenty-five patients were enrolled between August 2020 and August 2022 with median follow-up of 19.6 months. The pretreatment pathologic stages were high-grade (HG) T1 with carcinoma in situ (CIS; n = 7), HGT1 without CIS (n = 6), HGTa (n = 9), and CIS alone (n = 3). The 3-month complete response rate was 100% and recurrence-free survival at 12 months was 92%. Two patients with pretreatment HGT1 had HGT1 recurrences at 9 and 12 months. No patients progressed to T2 disease, underwent radical cystectomy, or had any radiographic evidence of progressive disease. Grade 1 AEs were common (23/25 patients) including hematuria, urinary frequency, urgency, and fatigue. Five patients (20%) experienced a grade 3 AE including hematuria and UTI.
Conclusions: In this single-arm phase 2 trial, GemDoce was well tolerated with promising efficacy for patients with BCG-naïve high-risk NMIBC.
Sunil H Patel,1 Andrew T Gabrielson,1 Sin Chan,1 Deborah Schwartz,1 Connie Collins,1 Nirmish Singla,1 Bruce Trock,1 Trinity J Bivalacqua,2 Noah Hahn1,3 Max R Kates1
- James Buchanan Brady Urological Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland
- Department of Urology, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania
- Greenberg Bladder Cancer Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland