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Table 4 Clinical trials with CSF1R inhibitors for the treatment of dt-GCT

From: Colony-stimulating factor 1 receptor (CSF1R) inhibitors in cancer therapy

Class Target Compound Clinical phase Sponsor Indication identifier Status/ Results Reference
Small molecules CSF1R (and cKIT, Flt3) Pexidartinib (PLX3397, PLX108-01) 1 Plexxikon Solid tumors and extension for MEC, dt-GCT, GIST, ATC, metastatic solid tumors NCT01004861 Ongoing
ORR: 12/23 (52%)
CBR: 19/23 (83%)
3 Plexxikon/Daiichi Sankyo dt-GCT or GCT-TS NCT02371369 Ongoing -
Monoclonal antibodies CSF1R Emactuzumab (RG7155) 1 Roche Solid tumors and dt-GCT NCT01494688 ORR: 24/28 (86%)
CBR: 27/28 (96%
CSF1R Cabiralizumab (FPA008) 1/2 FivePrime dt-GCT NCT02471716 Ongoing -
CSF1 MCS110 2 Novartis dt-GCT or GCT-TS NCT01643850 Ongoing
ORR: 4/4 (100%)
  1. ATC anaplastic thyroid cancer, CBR clinical benefit rate, CSF1 colony-stimulating factor 1, CSF1R colony-stimulating factor 1 receptor, dt-GCT diffuse-type tenosynovial giant cell tumor, GCT-TS giant cell tumor of the tendon sheath, GIST gastrointestinal stromal tumor, MEC mucoepidermoid carcinoma of the lung, ORR objective response rate