Table 2.
Ongoing trials of targeted therapies.
| Gene Alteration | Drug | Number of Patients Planned to Accrue | Primary Outcome | NCT Number |
|---|---|---|---|---|
| FGFR aberrations | Erdafitinib | 631 | OS | NCT03390504 (THOR) |
| TSC1/TSC2 mutations | Sapanisertib | 209 | ORR | NCT03047213 |
| Unselected | Buparlisib | 19 | 2-months PFS; PFS in the expansion cohort |
NCT01551030 |
| Unselected | Nivolumab + nabrapamycin | 34 | Maximum tolerated dose | NCT03190174 |
| Unselected | Nivolumab + IPI-549 | 160 | ORR | NCT03980041 (MARIO-275) |
| Unselected | Paclitaxel + sapanisertib | 52 | ORR | NCT03745911 |
| HER2 overexpressed | Trastuzumab deruxtecan + nivolumab | 99 | Part 1: dose-limiting toxicity Part 2: ORR |
NCT03523572 |
| EGFR, HER2, VEGFR, FGFR1/2, MET |
Afatinib Regorafenib Cabozantinib |
100 | ORR | NCT02795156 |
| ERBB1, ERBB2, ERBB3 | Afatinib | 42 | 6-months PFS | NCT02780687 |
| HER2-negative | RC48-ADC | 19 | ORR | NCT04073602 |
| HER2-positive | RC48-ADC | 60 | ORR | NCT03809013 |
| HER2-positive | PRS-343 | 85 | Incidence and severity of adverse events | NCT03330561 |
| HER2-positive | PRS-343 + atezolizumab | 45 | Incidence of dose-limiting toxicities; recommended phase 2 dose |
NCT03650348 |
| DDR genes | Olaparib | 30 | ORR | NCT03448718 |
| DDR genes | Olaparib | 60 | ORR | NCT03375307 |
| ARID1A, ATM | Olaparib + AZD6738 | 68 | ORR | NCT03682289 |
| Unselected | Niraparib + cabozantinib | 20 | Maximum tolerated dose; PFS |
NCT03425201 |
| Unselected | Niraparib | 58 | PFS | NCT03945084 |
| Unselected | Durvalumab + olaparib | 154 | PFS | NCT03459846 |
| Unselected | Atezolizumab + enfortumab vedotin; Atezolizumab + niraparib; Atezolizumab + Hu5F9-G4; Atezolizumab + tiragolumab; Atezolizumab + sacituzumab govitecan; Atezolizumab + tocilizumab; Atezolizumab + RO7122290 |
645 | ORR | NCT03869190 (MORPHEUS-UC) |
| BRCA1, BRCA2, PALB2, RAD51C, RAD51D | Rucaparib + lucitanib; Rucaparib + sacituzumab govitecan |
329 | Phase 1b: Safety and tolerability; Dose-limiting toxicityPhase 2: ORR |
NCT03992131 (SEASTAR) |
ADC, antibody-drug conjugate; ORR, overall response rate, OS, overall survival, PFS, progression-free survival.