Clinical trials with CSF1/CSF1R inhibitors in combination with anti-tumor therapies (excluding cancer-immunotherapy doublets)

ClassTargetCompoundCombination partnerClinical phaseSponsorIndicationClinicalTrials.gov identifierStatus/ResultsReference
Small moleculesCSF1R (and cKIT, Flt3)Pexidartinib(PLX3397, PLX108-01)Androgen-deprivation therapy plus external radiotherapy1Plexxikon/Daiichi SankyoProstate cancerNCT02472275Ongoing-
Paclitaxel1Plexxikon/Daiichi SankyoSolid tumorsNCT01525602ORR: 4/23 (17%)CBR: 14/23 (61%)[35]
Eribulin1/2Plexxikon/Daiichi SankyoBreast cancerNCT01596751Ongoing-
Temozolomide plus external radiotherapy1/2Plexxikon/Daiichi SankyoGBMNCT01790503Not yet reported-
Vemurafenib1Plexxikon/Daiichi SankyoBRAF-mutated melanomaNCT01826448Terminated-
PLX9486 (KIT inhibitor)1/2Plexxikon/Daiichi SankyoGISTNCT02401815Ongoing-
Sirolimus1/2Plexxikon/Daiichi SankyoAdvanced sarcomas, MPNSTNCT02584647Ongoing, not yet reported[73]
Monoclonal antibodiesCSF1REmactuzumab (RG7155)Paclitaxel1RocheOvarian and breast cancerNCT01494688Not yet reported-
CSF1REmactuzumab (RG7155)Paclitaxel plus bevacizumab2RocheOvarian cancerNCT02923739Ongoing-
CSF1MCS110Carboplatin plus gemcitabine2NovartisTNBCNCT02435680Ongoing-
CSF1PD-0360324Cyclophosphamide2PfizerRecurrent platinum-resistant epithelial ovarian, peritoneal, or fallopian tube cancerNCT02948101Ongoing-

CBR clinical benefit rate, CSF1 colony-stimulating factor 1, CSF1R colony-stimulating factor 1 receptor, dt-GCT diffuse-type tenosynovial giant cell tumor, GBM glioblastoma, GIST gastrointestinal stromal tumor, MPNST malignant peripheral nerve sheath tumor, ORR objective response rate, TNBC triple-negative breast cancer