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.