研究者情報

キクチ　エイジ   KIKUCHI EIJI   菊地栄次    所属   医学部医学科 腎泌尿器外科学    職種   主任教授
言語種別	日本語
発表タイトル	Efficacy and Safety of Nadofaragene Firadenovec for BCG-Unresponsive Non-Muscle-Invasive Bladder Cancer: Initial Results From an Ongoing Japanese Phase 3 Trial
会議名	第112回日本泌尿器科学会総会
学会区分	全国規模の学会
発表形式	口頭
講演区分	シンポジウム・ワークショップ・パネル（その他）
発表者・共同発表者	Inoue Keiji, Kikuchi Eiji, Nishiyama Hiroyuki, Nasu Yasutomo, Azuma Haruto, Eto Masatoshi, Falahati Ali, Hansen Sara Tantholdt, Juul Kristian, Fujimoto Kiyohide
発表年月日	2025/04/19
開催地 （都市, 国名）	福岡県福岡市
開催期間	2025/04/17～2025/04/19
概要	Objectives: Nadofaragene firadenovec-vncg is a replication-deficient recombinant adenoviral vector gene therapy carrying the IFNα2b transgene for high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC). A phase 3 trial (NCT05704244) was initiated in Japan to assess nadofaragene firadenovec efficacy and safety in Japanese participants with HG BCG-unresponsive NMIBC in 2 cohorts: CIS ± HG Ta/T1 and HG Ta/T1 only. We report initial results from participants with CIS ± HG Ta/T1. Methods: Participants received nadofaragene firadenovec intravesically once quarterly without reinduction in the absence of HG recurrence as assessed by cytology, cystoscopy, and biopsies if clinically indicated. Mandatory biopsies were conducted at month 12. The primary endpoint was complete response (CR) at any time after the first dose. Safety endpoints included the type, incidence, relatedness, and severity of treatment-emergent adverse events (AEs). Results: Twenty participants with CIS ± HG Ta/T1 were enrolled and initiated nadofaragene firadenovec treatment. Fifteen (75%) achieved a CR at month 3. In the safety analysis set, 76 study drug-related AEs were reported in 16 (80.0%) participants: 64 were grade 1 (84.2%) in 15 participants and 12 were grade 2 (15.8%) in 5 participants. No grade 3/4/5 AEs were reported. Conclusions: In this phase 3 trial, the 3-month efficacy of nadofaragene firadenovec appears to be higher than previously reported, with a clinically manageable and tolerable safety profile in Japanese participants.