Focus on current and emerging treatment options for glioma: A comprehensive review

Table 2 List of Immunotherapy

Ref.	Completion year	Demographics	Study phase	Identifier	Experimental drug	Sample size	Primary endpoint/outcomes	Results for primary outcome
BRAF/MEK inhibitors
Nicolaides et al[107], 2020	2023	Pediatrics	Phase 2	NCT01748149 (Ongoing Trial)	Vemurafenib	40	Safety and pharmacokinetics	Not yet reported
Hargrave et al[108], 2019	2020	Pediatrics	Phase 1/2a	NCT01677741	Dabrafenib	32	Objective response rates and safety	Objective response rate was 44% and 91% experienced adverse effects
Kaley et al[109], 2018	2016	Adults	Phase 2	NCT01524978	Vemurafenib	24	Confirmed objective response rate, PFS, OS and safety	Confirmed objective response rate was 25% and median PFS was 5.5 months
FGFR inhibitors
Lassman et al[110], 2022	2018	Adults	Phase 2	NCT01975701	Infigratinib	26	6-month PFS	6-month PFS rate was 16.0%
Bahleda et al[111], 2019	2017	Adults	Phase 1	NCT01703481	Erdafitinib	187	Safety	Most common treatment-related adverse events were hyperphosphatemia, dry mouth, and asthenia, generally grade 1/2 severity
HDAC inhibitors
Wood et al[112], 2018	2018	Pediatrics	Phase 1	ACTRN12609000978268	Panobinostat	9	Safety and pharmacokinetics	2 patients experienced Grade 3-4 thrombocytopenia, 1 experienced Grade 3 anemia, and 2 experienced Grade 3 neutropenia
Imipridone
Arrillaga-Romany et al[113], 2020	2023		Phase 2	NCT02525692 (Ongoing Trial)	ONC201	89	6-month PFS	Not yet reported
PI3K/mTOR inhibitors
Wen et al[114], 2022	2023	Adults	Phase 2	NCT03522298	Paxalisib	32	Safety and pharmacokinetics	Well-tolerated with adverse events consistent with other PI3K inhibitors
Wen et al[115], 2020	2020	Adults	Phase 1	NCT01547546	GDC-0084	47	Safety and pharmacokinetics	Well-tolerated with adverse events consistent with other PI3K inhibitors
Franz et al[116], 2015	2014	Adults/Pediatrics	Phase 1/2	NCT00411619	Enviroximes	28	6-month change in the volume of sub ependymal giant-cell astrocytoma	Statistically significant reduction in the volume of the primary sub ependymal giant-cell astrocytoma at 6 months
NTRK/ALK inhibitors
NCT02637687[117]	2026	Pediatrics	Phase 1/2	NCT02637687 (Ongoing Trial)	Larotrectinib	155	Objective response rates	Not yet reported
NCT02576431[118]	2025	Adults/Pediatrics	Phase 2	NCT02576431 (Ongoing Trial)	Larotrectinib	204	Objective response rates, PFS, OS, Safety	Not yet reported
Desai et al[119], 2022	2025	Adults/Pediatrics	Phase 1/2	NCT02650401 (Ongoing Trial)	Entrectinib	69	Maximum Tolerated Dose and Objective response rates	Not yet reported
IDH inhibitors
NCT05588141[120]	2029	Adults	Phase 1/2	NCT05588141 (Ongoing Trial)	Zotiraciclib	96	12-months PFS	Not yet reported
Mellinghoff et al[121], 2023	2027	Adults	Phase 3	NCT04164901	Vorasidenib	340	PFS	Significantly improved PFS
Mellinghoff et al[122], 2019	2024	Adults	Phase 1	NCT03343197	AG-120, AG881	49	2-hydroxyglutarate concentrationin resectedtumors	decreased tumorcell proliferationand immune cellactivation
EGFR inhibitors
Weller et al[123], 2017	2016	Adults	Phase 3	NCT01480479	Rindopepimut/Temozolomide	745	OS	Median OS was 20.1 months in the Rindopepimut group versus 20.0 months in the control group
Lassman et al[124], 2023	2022	Adults	Phase 3	NCT02573324	Depatuxizumab mafodotin	691	OS	No OS benefit for depatux-m in treating EGFR-amp newly diagnosed GBM

PFS: Progression-free survival; OS: Overall survival.