TABLE 2.
Clinical trials.
| NCT | Phase | N | Random | Drugs | Patient selection | Recruiting state | Results |
| RAS | |||||||
| NCT03979651 | Ib/II | 29 | NO | Trametinib plus hydroxychloroquine | activating NRAS mutation | Recruiting | – |
| NCT03973151 | I/II | 54 | NO | HL-085 (MEK inhibitor) | NRAS mutation | Recruiting | – |
| NCT01763164 | III | 402 | YES | MEK162 vs. dacarbazine | NRAS Q61 mutation | Completed | Dummer et al., 2017 |
| NCT01320085 | II | 183 | NO | MEK162 | BRAF V600 or NRAS Mutations | Active, not recruiting | Ascierto et al., 2013 |
| NCT01693068 | II | 194 | YES | Pimasertib vs. dacarbazine | NRAS mutation | Completed | Lebbe et al., 2016 |
| NCT03932253 | I | 37 | NO | FCN-159 | NRAS mutation | Recruiting | – |
| NCT01781572 | Ib/II | 102 | NO | LEE011 + MEK162 | NRAS mutation | Completed | Sosman et al., 2014 |
| NCT04109456 | I | 52 | NO | IN10018/IN10018 + Cobimetinib | uveal melanoma (UM); NRAS mutation | Recruiting | – |
| NCT02974725 | Ib | 195 | NO | LXH254 + LTT462/Trametinib/Ribociclib | NRAS mutation | Recruiting | – |
| NCT00866177 | II | 167 | NO | AZD6244 (Selumetinib) | V600E or V600K BRAF mutation; NRAS mutation at codons 12, 13, or 61 | Completed | Catalanotti et al., 2013 |
| NCT00338130 | II | 239 | YES | AZD6244/Temodal | BRAF and/or NRAS mutation | Completed | Kirkwood et al., 2012 |
| NCT03415126 | I | 49 | NO | ASN007 | BRAF mutation or fusion/NRAS and HRAS mutation/MEK1 mutation | Active, not recruiting | – |
| NCT03989115 | Ib/II | 144 | NO | RMC-4630+ Cobimetinib | KRAS mutations and amplifications, BRAF Class 3 mutations, or NF1 LOF mutations | Recruiting | – |
| NCT02065063 | I/II | 28 | NO | Trametinib + Palbociclib | BRAF V600 wild type and either NRAS wild type or NRAS mutation type | Completed | – |
| NCT03634982 | I | 240 | NO | RMC-4630 | RTK mutations, amplifications or rearrangements, KRASG12, BRAF Class 3, or NF1 LOF mutations | Recruiting | – |
| NCT04284774 | II | 49 | NO | Tipifarnib | HRAS genomic alterations | Not yet recruiting | – |
| NCT01941927 | II | 20 | NO | Trametinib + GSK2141795 (AKTi) | NRAS mutation or NRAS Wild-type/BRAF Wild-type. | Completed | Algazi et al., 2018 |
| NCT04059224 | II | 58 | no | Trametinib | BRAF V600 wild-type/NRAS wild-type/NRAS mutation | Recruiting | – |
| NCT04198818 | I/II | 150 | no | HH2710 | RAS/RAF/MEK/ERK mutation | Recruiting | – |
| NCT02857270 | I | 272 | no | LY3214996/LY3214996 + Midazolam/Abemaciclib/Nab-paclitaxel + Gemcitabine/Encorafenib + Cetuximab/ | Activating MAPK pathway alteration, BRAF mutation, NRAS mutation | Recruiting | – |
| NCT01390818 | I | 146 | NO | Pimasertib (MEKi) + Voxtalisib (PI3K/mTOR) | Genetic alteration in PTEN, BRAF, KRAS, NRAS, PI3KCA, ERBB1, ERBB2, MET, RET, KIT, GNAQ, GNA11 | Completed | Schram et al., 2018 |
| NF1 | |||||||
| NCT02465060 | II | 6452 | NO | Trametinib (MEKi) Defactinib (FAKi) | NF1 mutation, NF2 inactivating mutation | Recruiting | – |
| NCT03634982 | I | 240 | NO | RMC-4630 (SHP2i) | NF1 LOF | Recruiting | – |
| NCT03989115 | Ib/II | 144 | no | RMC-4630 + Cobimetinib | KRAS mutations and amplifications, BRAF Class 3 mutations, or NF1 LOF mutations | Recruiting | – |
| CDKN2A/CDK4 | |||||||
| NCT02478320 | II | 12 | NO | Ilorasertib (Aurora A/B/Ci) | CDKN2A deletion or mutation | Active, not recruiting | – |
| NCT02202200 | I/II | 40 | NO | Palbociclib (CDK4/6i) | BRAF V600E/K mutation, CDNKN2A loss and expression of Rb | Unknown | – |
| NCT03454919 | II | 60 | NO | Palbociclib (CDK4/6i) | Any gene aberrations in cell cycle pathways, including CDK4 amplification and/or CCND1 amplification and/or CDKN2A loss | Not yet recruiting | – |
| NCT02671513 | I | 30 | NO | SHR6390 (CDK4/6i) | Any cell cycle pathway abnormality (e.g., CDK4 amplify and/or CCND1 amplify and/or CDKN2A loss) | Unknown | – |
| NCT00835419 | II | 12 | NO | P276-00 (CDK1/4/9i) | cyclin D1 expression | Completed | – |
| NCT02065063 | I/II | 28 | NO | Trametinib + Palbociclib | BRAF V600 wild type and either NRAS wild type or NRAS mutation type | Completed | – |
| NCT01037790 | II | 304 | NO | Palbociclib (CDK4/6i) | CCND1 amplification, CDK4/6 mutation, CCND2 amplification OR any other functional alteration at the G1/S checkpoint. | Completed | – |
| NCT02308020 | II | 162 | NO | Abemaciclib | Any* | Completed | – |
| NCT01781572 | Ib/II | 102 | NO | LEE011 (ribociclib) + MEK162 | NRAS mutation | Completed | Sosman et al., 2014 |
| NCT02974725 | I | 315 | NO | LXH254 + LTT462; LXH254 + Trametinib; LXH254 + Ribociclib | NRAS mutation | Recruiting | – |
| PTEN | |||||||
| NCT03131908 | I/II | 41 | NO | GSK2636771 (Pi3Ki) + pembrolizumab | Evidence of PTEN loss | Recruiting | – |
| NCT01390818 | I | 146 | NO | Pimasertib (MEKi) + Voxtalisib (PI3K/mTORi) | Genetic alteration in PTEN, BRAF, KRAS, NRAS, PI3KCA, ERBB1, ERBB2, MET, RET, KIT, GNAQ, GNA11 | Completed | Schram et al., 2018 |
| MAP2K1/MAP2K2 | |||||||
| NCT01364051 | I | 19 | NO | Cediranib Maleate + Selumetinib | Any* | Active, not recruiting | – |
| NCT01941927 | II | 20 | no | Trametinib + GSK2141795 (AKTi) | BRAF Wild-type and NRAS mutations | Completed | Algazi et al., 2018 |
| NCT00948467 | I | 51 | NO | TAK-733 (MEKi) | Any* | Completed | Adjei et al., 2017 |
| NCT02296112 | II | 9 | NO | Trametinib | BRAF mutations in loci other than V600 (BRAF non V600 MUT) or BRAF fusion | Active, not recruiting | – |
| NCT02857270 | I | 272 | no | LY3214996/LY3214996 + Midazolam/Abemaciclib/Nab-paclitaxel + Gemcitabine/Encorafenib + Cetuximab/ | Activating MAPK pathway alteration, BRAF mutations, NRAS mutations | Recruiting | – |
| NCT04198818 | I/II | 150 | no | HH2710 | RAS/RAF/MEK/ERK mutations | Recruiting | – |
| NCT04059224 | II | 58 | no | trametinib | BRAF V600 wild-type/NRAS wild-type/NRAS mutant | Recruiting | – |
| KIT | |||||||
| NCT02501551 | II | 36 | NO | Regorafenib | KIT mutations | Recruiting | – |
| NCT01028222 | II | 55 | NO | Nilotinib/DTIC | KIT mutations | Completed | Guo et al., 2017 |
| NCT01395121 | II | 29 | NO | Nilotinib | KIT mutations | Completed | – |
| NCT01168050 | II | 25 | NO | Nilotinib | KIT mutations or amplification | Unknown | Carvajal et al., 2015 |
| NCT00470470 | II | 30 | NO | Imatinib mesylate | KIT mutations | Completed | Carvajal et al., 2011 |
| NCT00881049 | II | 1 | NO | Imatinib | KIT mutations | Completed | – |
| NCT01782508 | II | 40 | YES | Imatinib | KIT mutations | Unknown | – |
| NCT00577382 | II | 52 | NO | Sunitinib | KIT mutations | Completed | Buchbinder et al., 2015 |
| NCT00424515 | II | 24 | NO | Imatinib | KIT mutations | Completed | Hodi et al., 2013 |
| NCT00631618 | II | 12 | NO | Sunitinib | KIT mutations | Completed | – |
| NCT01738139 | I | 96 | NO | Ipilimumab + Imatinib | KIT mutations | Recruiting | – |
| NCT00700882 | II | 81 | NO | Dasatinib | KIT mutation or amplification | Active, not recruiting | – |
| NCT01390818 | I | 146 | NO | Pimasertib (MEKi) + Voxtalisib (PI3K/mTORi) | Genetic alteration in PTEN, BRAF, KRAS, NRAS, PI3KCA, ERBB1, ERBB2, MET, RET, KIT, GNAQ, GNA11 | Completed | Schram et al., 2018 |
| FBXW7 and RB1 | |||||||
| NCT02873975 | II | 50 | NO | LY2606368 (prexasertib) | MYC amplification, CCNE1 amplification, Rb loss, FBXW7 mutation, or another genomic abnormality indicative of replicative stress | Active, not recruiting | – |
| NCT02202200 | I/II | 40 | NO | Palbociclib (CDK4/6i) | BRAF V600E/K mutation, CDNKN2A loss and expression of Rb | Unknown | – |
| PI3KCA | |||||||
| NCT01449058 | Ib | 139 | NO | BYL719 + MEK162 | Any* | Completed | Juric et al., 2014 |
| NCT02646748 | I | 159 | NO | Pembrolizumb + Itacitinib (JAKi − INCB039110; Pembrolizumab + Parsaclisib (Pi3kδi INCB050465) | Any* | Active, not recruiting | – |
| NCT04282018 | I/II | 150 | no | BGB-10188/BGB-10188 + Zanubrutinib/BGB-10188 + Tislelizumab | Any* | Recruiting | – |
| NCT01390818 | I | 146 | NO | Pimasertib (MEKi) + Voxtalisib (PI3K/mTOR) | Genetic alteration in PTEN, BRAF, KRAS, NRAS, PI3KCA, ERBB1, ERBB2, MET, RET, KIT, GNAQ, GNA11 | Completed | Schram et al., 2018 |
| WT1 | |||||||
| NCT03311334 | Ib/II | 84 | NO | DSP-7888 + Nivolumab/DSP-7888 + Pembrolizumab | Any* | Recruiting | |
| NCT02498665 | I | 24 | NO | DSP-7888 | Any* | Completed | |
| MITF | |||||||
| NCT01065467 | I | 16 | NO | LBH589 | Any* | Completed | Ibrahim et al., 2016 |
| MTOR | |||||||
| NCT01960829 | II | 60 | NO | Everolimus | MTOR mutation | Unknown | |
| NCT01166126 | II | 4 | NO | Temsirolimus/AZD 6244 | BRAF mutation | Terminated | |
| NCT01014351 | II | 70 | NO | Everolimus + paclitaxel + carboplatin | Any | Completed | Hauke et al., 2013 |
| NCT01522820 | I | 18 | NO | Rapamycin + dendritic Cell Vaccine | NY-ESO-1 expression | Completed | |
| NCT00655655 | I | 96 | NO | Everolimus + Vatalanib | Any | Completed | Zhu et al., 2020 |
| NCT01596140 | I | 27 | NO | Vemurafenib + Everolimus + Temsirolimus | Any | Completed | |
| NCT02201212 | II | 30 | NO | Everolimus | TSC1 or TSC2 mutation or activating MTOR mutations | Completed | |
*Any: population not selected for mutation. Clinicaltrials.gov accessed May 10, 2020.