Recent and ongoing clinical trials investigating agents targeting the key driver mutations in CCA

TargetTargeted agentsStudyPatient population (n)StatusResultsNCT number
IDH1/2IvosidenibPhase 1, multicenter, open-label73Active, not recruitingOngoingNCT02073994
Phase 3, multicenter, (ClarIDHy)185Active, not recruitingmPFS = 2.7 mos (ivosidenib) vs. mPFS = 1.4 mos (placebo)NCT02989857
FT2012Phase 1/2200 (estimated)Active, not recruitingOngoingNCT03684811
BAY1436032Phase 1, open-label, non-randomized, multicenter81Active, not recruitingOngoingNCT02746081
FGFR2PemigatinibPhase 2, open-label, single-arm, multicenter study (FIGHT-202)146Active, not recruitingORR = 35.5%; PFS = 6.9 mos; DoR = 7.5 mos (interim results for 107 pts)NCT02924376
Phase 3, open-label, randomized, active-controlled, multicenter (FIGHT-302)432 (estimated)RecruitingOngoingNCT03656536
InfigratinibPhase 2, single arm160 (estimated)RecruitingORR = 14.8%; DCR = 75.4%; mPFS = 5.8 mos (interim results for 61 pts)NCT02150967
Phase 3 multicenter, open-label, randomized (The PROOF Trial)384 (estimated)RecruitingOngoingNCT03773302
DerazantinibPhase 2, open-label, single-arm study143 (estimated)RecruitingOngoingNCT03230318
FutinatinibPhase 1/2386Active, not recruitingORR = 37.3%; DCR = 82.1%; mPFS = 7.2 mos; mDoR = 6.2 mos (interim results)NCT02052778
Phase 3, open-label, randomized (FOENIX-CCA3)216 (estimated)RecruitingOngoingNCT04093362
ErdatifinibPhase 235Active, not recruitingOngoingNCT02699606
Debio-1347Phase 2 basket (FUZE Clinical Trial)63Active, not recruitingOngoingNCT03834220
PonatinibPhase 245 (estimated)RecruitingOngoingNCT02272998
NTRKEntrectinibPhase 2 basket, open-label, multicenter, global (STARTRK-2)700 (estimated)RecruitingOngoingNCT02568267
LarotrectinibPhase 2 basket (NAVIGATE)203 (estimated)RecruitingOngoingNCT02576431
BRAFDabrafenib + trametinibPhase 2, open-label206Active, not recruitingORR = 47%; mDoR ≥6 mos in the 54% of responders; PFS = 7.2 mos; OS = 11.3 mos (interim results for 43 pts)NCT02034110

mPFS: median progression-free survival; DCR: disease control rate; mos: months; ORR: overall response rate; DoR: duration of response; mDoR: median duration of response; pts: patients