Ongoing first-line phase III trials with targeted agents for advanced BTCs
| Study name/number | Target population | Estimated sample size | Experimental treatment | Comparator | Primary endpoint | Secondary endpoints |
|---|---|---|---|---|---|---|
| FIGHT-302 | FGFR2 rearrangements | 432 patients | Pemigatinib13.5 mg QD on a 3-week cycle | Cisplatin 25 mg/mq + gemcitabine1000 mg/mq on days 1 and 8 q3w up to 8 cycles | PFS per RECIST v1.1 by ICR | OS, ORR, DOR, DCR per RECIST v1.1 by ICR, safety, QoL |
| PROOF trialNCT03773302 | FGFR2 fusions | 384 patients | Infigratinib 125 mg orally QD, 3 weeks on, 1 week off | Cisplatin 25 mg/mq + gemcitabine1000 mg/mq on days 1 and 8 q3w up to 8 cycles | PFS per RECIST v1.1 by ICR | OS, ORR, DOR, BOR, DCR per RECIST v1.1 by ICR, PFS per investigator assessment, safety |
| FOENIX-CCA3 | FGFR2 rearrangements | 216 patients | Futibatinib 20 mg orally QD on a 3-week cycle | Cisplatin 25 mg/mq + gemcitabine1000 mg/mq on days 1 and 8 q3w up to 8 cycles | PFS per RECIST v1.1 by ICR | OS, ORR, DCR per RECIST v1.1 by ICR, PFS per investigator assessment, safety |
Crossover is allowed in FIGHT-302 and FOENIX-CCA3; QD: once a day; q3w: every 3 weeks; ICR: independent central review; QoL: quality of life; BOR: best overall response