Table 1.

Phase II/III clinical trials with immune checkpoint inhibitors in glioblastoma.

Clinical Trial	Phase	Target	Treatment	Control	Indication	# Patients	Endpoint	Outcome
CheckMate 143 NCT02017717 PMC5463583	III	PD-1	Nivolumab	Bevacizumab	R GB	Each arm: ~185	OS	No impact
CheckMate 498 NCT02617589	III	PD-1	RT, nivolumab	SOC	P GB MGMT-unmeth.	Each arm: ~275	OS	No impact
CheckMate 548 NCT02667587	II	PD-1	SOC, nivolumab	SOC, placebo	P GB MGMT-meth.	Each arm: ~160	PFS	No impact
Neo-nivo NCT02550249 30742120	II	PD-1	Nivolumab (neo-adjuvant), (S)S, nivolumab (adjuvant)	None	P GB R GB	P: 3 R: 27	(OS)	7.3 mo
NCT02337491	II	PD-1	Pembrolizumab, bevacizumab	Pembrolizumab	R GB	Treatment: 50 Control: 30	(OS)	8.8 mo vs. 10.3 mo
NCT02337686	II	PD-1	SS, pembrolizumab	None	R GB	15	PFS6	53%
NCT02852655 30742122	II	PD-1	Pembrolizumab (neo-adjuvant), SS, pembrolizumab (adjuvant)	SS, pembrolizumab (adjuvant)	R HGG	Treatment: 16 Control: 19	(OS)	13.7 mo vs. 7.5 mo
NCT03291314	II	PD-L1	Avelumab, axitinib	None	R GB	32	PFS6	18%
SEJ NCT03047473	II	PD-L1	SOC, avelumab	None	P GB	24	(PFS)	11.9 mo
NCT02336165	II	PD-L1	SOC, durvalumab	Historical	P GB MGMT-unmeth.	40	OS12	60% vs. 50%

Abbreviations: MO, month; #, number of; RT, radiotherapy; SOC, standard of care; SS, second surgical procedure; P, primary; R, recurrent; GB, glioblastoma; MGMT, O6-methylguanine-DNA methyltransferase gene; (un)meth., (un)methylated gene promoter; PFS6, 6-mo progression-free survival (PFS); OS12, 12-mo OS. Reference numbers (NCT, PMID, PMCID) corresponding to each clinical trial are indicated in the ‘Clinical trial’ column. The ‘Endpoint’ column indicates which primary survival endpoint was assessed in each clinical trial. If a study only used a secondary survival endpoint, the outcome measure was placed between parentheses.