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

Class	Target	Compound	Clinical phase	Sponsor	Indication	ClinicalTrials.gov 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	OngoingORR: 12/23 (52%)CBR: 19/23 (83%)	[22]
Small molecules	CSF1R (and cKIT, Flt3)	Pexidartinib (PLX3397, PLX108-01)	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%	[12]
	CSF1R	Cabiralizumab (FPA008)	1/2	FivePrime	dt-GCT	NCT02471716	Ongoing	-
	CSF1	MCS110	2	Novartis	dt-GCT or GCT-TS	NCT01643850	OngoingORR: 4/4 (100%)	[21]

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