FOENIX-CCA4: A phase 2 study of futibatinib 20 mg and 16 mg in patients with advanced cholangiocarcinoma (CCA) and fibroblast growth factor receptor 2 (FGFR2) fusions/rearrangements.

• DOI: 10.1200/jco.2024.42.3_suppl.tps572
• Publication Date: 2024-01-22T21:12:58Z
• Authors: Teresa Macarulla; Takashi Mizuno; Giovanni Brandi; Jin Li; Ming-Huang Chen; Jung Hun Kang; Kathia Cristina A...; Piotr Jan Wysocki; Nuno Couto; Bailey Anderson; Hamdy Elsayed; Parvez Mantry
• Keywords: Clinical endpoint

TPS572 Background: In 2022, the U.S. FDA approved futibatinib, a covalently binding inhibitor of FGFR1–4, for previously treated patients with advanced intrahepatic CCA and FGFR2 fusions/rearrangements. pivotal phase 2 FOENIX-CCA2 study, futibatinib 20 mg once daily (QD) resulted in an objective response rate (ORR) 42% by independent review median duration (DOR) 9.7 months. Futibatinib was tolerable had manageable safety profile. 1 Limited data suggest may also have antitumor activity at lower dose (16 QD), fewer modifications required adverse events. The aim FOENIX-CCA4is to confirm clinical benefit QD explore efficacy 16 Methods: this global, 2, randomized, open-label study (NCT05727176), harboring fusions/rearrangements who received ≥1 line gemcitabine/platinum-based therapy advanced/metastatic disease will be randomized 1:1 starting or continuously on 21-day cycle. Randomization stratified number prior lines systemic (1 vs ≥2). continued until progressive unacceptable toxicity. Tumor assessed baseline, every 6 weeks first 12 weeks, 9 thereafter radiographic initiation new anticancer therapy. primary endpoint is ORR according Response Evaluation Criteria Solid Tumors version 1.1, as blinded central (analyzed when all responding ≥6 months’ follow-up from onset response). DOR key secondary endpoint. Other endpoints are progression-free survival, overall safety, quality life. Sample size (120 patients) estimated assumption that if between 32% (with ≥60 per arm), probability correctly selecting treatment arm true 0.87. Enrollment ongoing Europe, US, Asia-Pacific. 1. Goyal L et al. N Engl J Med. 2023;388:228–39. 2. Meric-Bernstam F Cancer Discov. 2022;12:402–15. Clinical trial information: NCT05727176 .