Table 1.
Current Targeted Therapy Clinical Trials in Solid Organ Malignancies including Cholangiocarcinoma
| Target | Agent | Trial Description | NCT Number |
|---|---|---|---|
| FGFR | NVP-BGJ398 | Phase II trial in patients with advanced CCA harboring FGFR gene fusions/aberrations | NCT02150967 |
| FGFR | JNJ-42756493 | Phase I trial in patients with advanced solid organ malignancy or lymphoma | NCT01703481 |
| FGFR | ARQ 087 | Phase 1/II trial in patients with solid organ malignancy and FGFR genetic alterations (including iCCA patients with FGFR2 gene fusions) | NCT01752920 |
| FGFR | TAS-120 | Phase I trial in patients with advanced solid malignancy or multiple myeloma with or without FGF/FGFR-related abnormalities | NCT02052778 |
| FGFR | CH5183284/Debio 1347 | Phase I trial in patients with solid organ malignancy and genetic aberration of FGFR1, 2, or 3 | NCT01948297 |
| FGFR2 | Ponatinib | Phase II trial in patients with advanced cholangiocarcinoma harboring FGFR2 gene fusions | NCT02265341 |
| FGFR2 | FPA144 | Phase I trial in patients with advanced solid tumors with FGFR2b overexpression/amplification | NCT02318329 |
| IDH1 | AG-120 | Phase I trial in patients with advanced solid tumors harboring an IDH1 mutation | NCT02073994 |
| IDH1 | AG-120 | Phase III, multi-center, double-blind, placebo-controlled trial in patients with advanced cholangiocarcinoma harboring IDH1 mutations | NCT02989857 |
| MEK | Trametinib | Phase II trial in patients with advanced CCA or gallbladder cancer | NCT02042443 |
| MEK, VEGFR/PDGFR/Raf | GSK1120212 plus Pazopanib | Phase I trial in patients with advanced solid tumors | NCT01438554 |
| PD-1 | Pembrolizumab | Phase II trial in patients with advanced solid tumors | NCT02628067 |
| PD-1 | MK-3475 (Pembrolizumab) | Phase I/II trial in patients with advanced gastrointestinal tumors | NCT02268825 |