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.