TABLE 1

List of clinical trials for glioblastoma treatment registered on www.clinicaltrials.gov

Small molecules.

No.	Drug	Purpose	P	DR	NPE	AG	NCT number	Status
1	Abemaciclib	To evaluate the efficacy of abemaciclib in recurrent GBM	2	O	47	A,S	NCT02981940	Recruiting
2	ACP-196	To evaluate the efficacy and safety of ACP-196 in patients with recurrent GBM who have progressed after 1 or 2 prior systemic treatment regimens	1/2	O	72	A,S	NCT02586857	Recruiting
3	Afatinib	To determine the maximum safe dose of afatinib that can be administered to people with brain cancer	1	O	24	A,S	NCT02423525	Recruiting
4	Aldoxorubicin	To determine the efficacy and safety of aldoxorubicin in patients with GBM	2	i.v.	28	A,S	NCT02014844	Completed (Groves et al., 2016)
5	Alisertib	To study the side effects and best dose of alisertib when combined with fractionated stereotactic radiosurgery in treating patients with high-grade gliomas	1	O	24	A,S	NCT02186509	Active, not recruiting
6	AMG-232	To study the side effects and dosage of MDM2 inhibitor AMG-232 in patients with newly diagnosed or recurrent GBM	1	O	92	A,S	NCT03107780	Not yet recruiting
7	Ascorbate	To evaluate high-dose ascorbate in combination with standard of care treatment of GBM	2	i.v.	90	A,S	NCT02344355	Recruiting
8	Atorvastatin	To explore the efficacy and safety of atorvastatin in combination with RT+TMZ in patients with newly diagnosed GBM	2	O	32	A,S	NCT02029573	Completed
9	Axitinib	To test the efficacy of axitinib alone or in combination with lomustine for patients with recurrent GBM	2	O	52	A,S	NCT01562197	Completed (Duerinck et al., 2016)
10	Axitinib + Avelumab	To determine the efficacy of axitinib + avelumab to treat patients with recurrent GBM	2	O	52	A,S	NCT03291314	Recruiting
11	AZD1390	To test the safety and tolerability of AZD1390 in combination with radiation therapy for the treatment of brain tumors	1	i.v.	132	A,S	NCT03423628	Not yet recruiting
12	BAL101553	To assess side effects and best dose of BAL101553 + radiation therapy in patients with newly diagnosed GBM	1	O	30	A,S	NCT03250299	Recruiting
13	BBI608 (napabucasin)	To test the efficacy of BBI608 in combination with TMZ in patients with recurrent or progressive GBM	1/2	O	60	A,S	NCT02315534	Recruiting
14	Belinostat	To determine the efficacy of belinostat in patients with newly diagnosed GBM and to determine the feasibility of adding magnetic resonance spectroscopic imaging to improve patient outcomes	2	i.v.	87	A,S	NCT02137759	Recruiting
15	Bevacizumab + Nimustine	To determine the efficacy and feasibility of bevacizumab and nimustine treatment in patients with recurrent GBM	2	i.v.	40	A,S	NCT02698280	Recruiting
16	BGB-290	To assess the combination of BGB-290 and TMZ in patients with newly diagnosed or recurrent GBM	1/2	O	300	A,S	NCT03150862	Recruiting
17	BGJ398	To determine the efficacy of BGJ398 in patients with recurrent resectable or unresectable GBM	2	O	24	A,S	NCT01975701	Active, not recruiting
18	BLZ945	To characterize the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of BLZ945 against GBM	2	O	151	A,S	NCT02829723	Recruiting
19	Buparlisib	To test the efficacy of buparlisib plus carboplatin or lomustine in patients with recurrent GBM	1/2	O	35	A,S	NCT01934361	Completed
20	Cabazitaxel	To assess the efficacy of cabazitaxel on GBM	2	IF	24	A,S	NCT01866449	Active, not recruiting
21	Cabozantinib	To study the feasibility and efficacy of cabozantinib for recurrent or refractory GBM	2	O	10	C,A	NCT02885324	Recruiting
22	Capecitabine	To test the efficacy of capecitabine + bevacizumab in patients with recurrent GBM	1	O	12	A,S	NCT02669173	Recruiting
23	Cediranib + Olaparib	To evaluate to efficacy of cediranib + olaparib in patients with recurrent GBM	2	O	70	A,S	NCT02974621	Recruiting
24	Chlorogenic acid	To determine the pharmacokinetic characteristics of chlorogenic acid in advanced GBM	1	i.v.	30	A	NCT02728349	Recruiting
25	Chloroquine	To assess the safety of chloroquine addition to chemoradiation in newly diagnosed GBM	1	O	9	A,S	NCT02378532	Active, not recruiting
26	Crenolanib	To investigate crenolanib monotherapy in patients with recurrent/refractory GBM with PDGFRA gene amplification	2	O	33	A,S	NCT02626364	Recruiting
27	Crizotinib	To assess the safety, efficacy, and safety of crizotinib in combination with RT+TMZ in patients with newly diagnosed GBM	1	O	24	A,S	NCT02270034	Recruiting
28	Dacomitinib	To assess the efficacy and safety of dacomitinib in patients with recurrent GBM with EGFR gene amplification and/or EGFRvIII mutation.	2	O	64	A,S	NCT01520870	Active, not recruiting
29	Dexanabinol	To determine the maximum safe dose of dexanabinol that can be administered to people with brain cancer	1	i.v.	26	A,S	NCT01654497	Completed
30	Dimethyl fumarate	To test the safety of dimethyl fumarate in combination with RT+TMZ in patients with newly diagnosed GBM	1	O	12	A,S	NCT02337426	Active, not recruiting
31	Disulfiram	To assess the effects of proteasome inhibition in patients with GBM	1	O	20	A,S	NCT01907165	Active, not recruiting
32	DM-CHOC-PEN	To test the efficacy of DM-CHOC-PEN in patients with GBM	2	i.v.	27	A,S	NCT02038218	Completed
33	Dovitinib	To determine a safe and tolerable dose of dovitinib in patients with relapsed GBM	1	O	12	A,S	NCT01972750	Completed (Schäfer et al., 2016)
34	Dovitinib	To determine the efficacy of dovitinib on recurrent GBM	2	O	33	A,S	NCT01753713	Completed (Ahluwalia et al., 2015)
35	Epacadostat	To determine the efficacy of epacadostat in combination with nivolumab for patients with GBM	2	O	291	A,S	NCT02327078	Recruiting
36	Fingolimod	To evaluate the efficacy of fingolimod in patients with bevacizumab-resistant GBM	1	O	5	A,S	NCT02490930	Completed
37	G-202 (mipsagargin)	To evaluate the activity, safety, and CNS exposure of G-202 in patients with recurrent or progressive GBM	2	i.v.	26	A,S	NCT02067156	Completed, Publication Awaited
38	GDC-0084	To evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of GDC-0084 in patients with progressive or recurrent GBM	1	O	29	A,S	NCT01547546	Completed (Wen et al., 2016)
39	HMPL-813	To evaluate epitinib to treat GBM patients with EGFR gene amplification	1	O	29	A,S	NCT03231501	Not yet recruiting
40	INC280 + Buparlisib	To assess the safety of the combination of INC280 and buparlisib in patients with recurrent GBM	1/2	O	42	A,S	NCT01870726	Completed (van den Bent et al., 2017)
41	Indoximod	To assess the effect of indoximod in patients with newly diagnosed GBM	1/2	O	144	C,A,S	NCT02052648	Recruiting
42	Ixazomib	To determine the tissue concentration of ixazomib citrate	1	O	3	A,S	NCT02630030	Recruiting
43	JP001	To evaluate the effect of JP001 in combination with standard chemoradiation on increasing overall survival of patients with newly diagnosed GBM	2/3	O	264	A,S	NCT03008148	Not yet recruiting
44	Lapatinib	To test the safety and effects of a combination of lapatinib, plus RT+TMZ in patients with newly diagnosed GBM	2	i.v.	70	A,S	NCT01591577	Recruiting
45	LB100	To determine blood-brain barrier permeability of LB100	1	i.v.	20	A,S	NCT03027388	Not yet recruiting
46	LOXO-101	To determine the efficacy of LOXO-101 in the treatment of solid tumors	2	O	151	C,A,S	NCT02576431	Recruiting
47	LY2157299	To test the efficacy of LY2157299 in combination with lomustine in patients with recurrent GBM	2	O	180	A,S	NCT01582269	Active, not recruiting
48	LY2228820	To determine an appropriate dose of LY2228820 in combination with TMZ and radiotherapy in patients with newly diagnosed GBM	1/2	O	50	A,S	NCT02364206	Recruiting
49	Macitentan	To test the safety of macitentan in patients with newly diagnosed GBM	1	O	30	A,S	NCT02254954	Completed
50	Marizomib	To establish the impact of marizomib on overall survival of patients with GBM	3	i.v.	750	A,S	NCT03345095	Not yet recruiting
51	Marizomib	To determine the efficacy of marizomib in patients with newly diagnosed GBM	1	i.v.	48	A,S	NCT02903069	Recruiting
52	Mebendazole	To determine the safety and side effects for increasing doses of mebendazole for recurrent or progressive pediatric brain tumors	1	O	21	C,A	NCT02644291	Recruiting
53	Mibefradil	To determine the safety of mibefradil and hypofractionated re-irradiation therapy in recurrent GBM	1	O	24	A,S	NCT02202993	Completed
54	Nabiximols	To determine the safety of nabiximols in combination with TMZ in patients with recurrent GBM	1/2	O	6	A,S	NCT01812603	Completed (Twelves et al., 2017)
55	NVX-108	To test the safety, tolerability, and effectiveness of NVX-108	1	i.v.	25	A,S	NCT02189109	Active, not recruiting
56	Olaparib	To determine efficacy of olaparib in patients with glioma, cholangiocarcinoma, or solid tumors with IDH1 or IDH2 mutations	2	O	75	A,S	NCT03212274	Not yet recruiting
57	ONC201	To test efficacy of ONC201 in patients with recurrent GBM	2	O	30	A,S	NCT02525692	Recruiting
58	Ortataxel	To evaluate the efficacy of ortataxel in recurrent GBM	2	i.v.	64	A,S	NCT01989884	Suspended
59	Palbociclib Isethionate	To test the side effects and best dose of palbociclib isethionate in treating younger patients with central nervous system tumors	1	O	55	C,A	NCT02255461	Recruiting
60	Pazopanib	To assess pazopanib in combination with TMZ in patients with newly diagnosed GBM after surgery and RT-CT	1/2	O	51	A,S	NCT02331498	Recruiting
61	Pembrolizumab + Vorinostat + TMZ	To test the safety and tolerability of vorinostat and pembrolizumab, in combination with TMZ and radiotherapy	1	O	32	A,S	NCT03426891	Not yet recruiting
62	Perifosine + Torisel (Temsirolimus)	To test the effectiveness of perifosine and torisel in patients with recurrent or progressive GBM	2	O	10	A,S	NCT02238496	Active, not recruiting
63	Plerixafor	To determine the safety of plerixafor after radiation therapy and TMZ in patients with newly diagnosed GBM	1/2	O	29	A,S	NCT01977677	Active, not recruiting
64	PLX3397	To test the efficacy of PLX3397 in combination with radiation therapy (RT) + TMZ in patients with newly diagnosed GBM	1/2	O	65	A,S	NCT01790503	Active, not recruiting
65	Ponatinib	To evaluate the efficacy of ponatinib in recurrent GBM	2	O	32	A,S	NCT02478164	Active, not recruiting
66	PQR309	To evaluate the dual pan-PI3K and mTOR inhibitor in patients with first progression of GBM	2	O	>35	A,S	NCT02850744	Active, not recruiting
67	PT2385	To study efficacy of HIF-2 alpha inhibitor PT2385 in patients with recurrent GBM	2	O	35	A,S	NCT03216499	Recruiting
68	Regorafenib	To evaluate the role of regorafenib in prolonging the overall survival of GBM patients	2	O	112	A,S	NCT02926222	Active, not recruiting
69	Ribociclib	To assess the ability of ribociclib to inhibit CDK4/CDK6/RB/E2F signaling and cell proliferation/viability in core and infiltrating tumor tissues obtained from patients with recurrent GBM	1	O	20	A,S	NCT02345824	Recruiting
70	Sapanisertib	To determine blood-brain barrier permeability and efficacy of sapanisertib	1	O	40	A,S	NCT02133183	Recruiting
71	Sapanisertib	To determine the best dose of sapanisertib in combination with bevacizumab in patients with recurrent GBM or advanced solid tumors	1	O	23	A,S	NCT02142803	Recruiting
72	Selinexor	To evaluate the efficacy and safety of selinexor in patients with recurrent GBM	2	O	125	A,S	NCT01986348	Active, not recruiting
73	Sunitinib	To determine the effectiveness of a combination of sunitinib, TMZ, and RT in newly diagnosed GBM patients harboring tumors with unmethylated MGMT promoter	2	O	45	A,S	NCT02928575	Recruiting
74	Sunitinib	To evaluate the effect of high-dose, intermittent sunitinib in patients with recurrent GBM	2/3	O	100	A,S	NCT03025893	Not yet recruiting
75	Tesevatinib	To assess the efficacy of tesevatinib monotherapy in recurrent GBM	2	O	40	A,S	NCT02844439	Active, not recruiting
76	TG02	To determine safety and efficacy of TG02 in patients with recurrent GBM and anaplastic astrocytoma	1/2	O	152	A,S	NCT02942264	Recruiting
77	TH-302	To determine the safety and efficacy of TH-302 in combination with bevacizumab for GBM following bevacizumab failure	2	O	33	A,S	NCT02342379	Active, not recruiting
78	Tipifarnib	To test the safety and effectiveness of tipifarnib for newly diagnosed GBM	1	O	19	A,S	NCT02227901	Completed
79	Tivozanib	To test the safety and effectiveness of tivozanib for recurrent GBM	2	O	10	A,S	NCT01846871	Completed (Kalpathy-Cramer et al., 2017)
80	TPI 287	To evaluate the safety, maximum tolerated dose (MTD), and efficacy of TPI 287 in combination with bevacizumab in patients with GBM	2	i.v.	17	A,S	NCT02047214	Terminated
81	Trametinib	To investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation-positive relapsed or refractory GBM	2	O	40	C	NCT02684058	Recruiting
82	TRC102	To evaluate the combination of TRC102 and TMZ in patients with recurrent GBM	2	O	66	A,S	NCT02395692	Active, not recruiting
83	Ubidecarenone	To study the side effects and dosage of ubidecarenone injectable nanosuspension in patients with recurrent GBM or gliosarcoma	1	i.v.	10	A,S	NCT03020602	Recruiting
84	USL311	To assess the safety and efficacy of USL311 alone and in combination with lomustine in patients with relapsed or recurrent GBM	2	O	120	A,S	NCT02765165	Recruiting
85	VAL-083	To investigate the efficacy of VAL-083 in patients with TMZ-bevacizumab recurrent GBM	3	i.v.	180	AS	NCT03149575	Recruiting
86	VAL-083	To determine the efficacy of VAL-083 on unmethylated MGMT recurrent GBM	2	i.v.	48	A,S	NCT02717962	Recruiting
87	Veliparib	To determine efficacy of veliparib + TMZ in patients with newly diagnosed GBM	2/3	O	440	A,S	NCT02152982	Recruiting
88	Vistusertib	To determine the efficacy of mTORC1/2 kinase inhibitor vistusertib in previously treated GBM	1/2	O	52	A,S	NCT02619864	Recruiting
89	Vorinostat	To determine the efficacy of vorinostat + bevacizumab in patients with recurrent GBM	2	O	48	A,S	NCT01738646	Completed (Ghiaseddin et al., 2018)
90	β-elemene	To determine the efficacy of β-elemene to maintain the health of patients with newly diagnosed malignant gliomas following standard treatment	3	i.v.	100	A,S	NCT02629757	Recruiting

A, adult (18–60 years old); AG, age groups; C, child (<18 years old); CDK4/6, cyclin-dependent kinase 4/6; DR, delivery route; IF, infusion; i.v., intravenous; MDM2, mouse double minute 2 homolog; mTOR, mammalian target of rapamycin complex 1; NPE, number of patients enrolled; O, oral; P, phase; S, senior (>60 years old); TMZ, temozolomide.