Table 4.
Selected ongoing and upcoming clinical trials evaluating targeted therapies in patients with advanced biliary tract cancer *.
| Clinical Trials | Targeted Agent | Study Phase | Signaling Pathway Targeted | Primary Endpoint | Study Identifier (ClinicalTrials.gov Identifier) |
|---|---|---|---|---|---|
| First Line | Pemigatinib vs. gemcitabine+cisplatin | III | FGFR | PFS | NCT03656536 (FIGHT-302) |
| Infigratinib vs. gemcitabine+cisplatin | III | FGFR | PFS | NCT03773302 (PROOF) | |
| Futibatinib vs. gemcitabine+cisplatin | III | FGFR | PFS | NCT04093362 (FOENIX) | |
| Varlitinib + gemcitabine + cisplatin | IB/II | EGFR, HER2, HER4 | MTD, PFS | NCT02992340 | |
| Copanlisib + gemcitabine + cisplatin | II | mTOR | PFS | NCT02631590 | |
| Olaparib | II | DNA damage repair | ORR | NCT04042831 | |
| Second or Later Line | Derazantinib | II | FGFR | ORR, and PFS at 3 months | NCT03230318 (FIDES-01) |
| BGJ398 | II | FGFR | ORR | NCT02150967 | |
| Futibatinib | II | FGFR | ORR | NCT02052778 | |
| JNJ-42756493 (Erdafitinib) | II | FGFR | ORR | NCT02699606 | |
| Ivosidenib or pemigatinib in combination with Gemcitabine and cisplatin | I | FGFR and IDH1 | Tolerability | NCT04088188 | |
| Rucaparib + nivolumab | II | PARP + PD-1 | PFS rate at 4 months | NCT03639935 | |
| Olaparib | II | IDH mutation associated ‘BRCAness’ | ORR | NCT03212274 | |
| Niraparib | II | BAP1 and DDR pathway | ORR | NCT03207347 | |
| Entrectinib | II | NTRK, ROS1, or ALK | ORR | NCT02568267 | |
| PLX8394 | I/II | BRAF | Pharmacokinetics, ORR | NCT02428712 | |
| Gemcitabine + selumetinib vs. gemcitabine | II | MEK | ORR | NCT02151084 | |
| AG-881 | I | IDH | Safety, MTD, RP2D | NCT02481154 | |
| FT 2102 | Ib/II | IDH1 | MTD, RP2D, ORR | NCT03684811 | |
| BAY1436032 | I | IDH1 | Safety, MTD, RP2D | NCT02746081 | |
| Afatinib + capecitabine | I | EGFR | Safety, MTD, RP2D | NCT02451553 | |
| Trastuzumab deruxtecan (DS-8201) | II | HER2 | ORR | JMA-IIA00423 (HERB) | |
| KA2507 | Ib/II | HDAC | MTD, PFS rate at 4 months | NCT04186156 | |
| Fruquintinib | II | VEGFR | PFS | NCT04156958 |
Abbreviations: PFS, progression-free survival; MTD, maximal tolerated dose; ORR, overall response rate; RP2D, recommended phase II dose. * Information obtained from ClinicalTrials.gov (https://clinicaltrials.gov/), accessed between 17 May 2020, and 21 June 2020.