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. Author manuscript; available in PMC: 2021 Jan 1.
Published in final edited form as: Nat Rev Cancer. 2019 Dec 5;20(1):12–25. doi: 10.1038/s41568-019-0224-7

Table 1.

Ongoing clinical trials in brain tumours

Therapy Cancer type Phase NCT identifier
Personalized neoepitope-based vaccine Recurrent paediatric brain tumours I NCT03068832 (ref.[211])
Personalized neoantigen DNA vaccine in combination with immune checkpoint blockade therapy Newly diagnosed, unmethylated glioblastoma I NCT04015700 (ref.[212])
IDO inhibitor (indoximod) in combination with the chemotherapy temozolomide Progressive primary malignant paediatric brain tumours I NCT02502708 (ref.[53])
Anti-LAG3 +/− anti-PD1 therapy nivolumab Recurrent glioblastoma I NCT02658981 (ref.[168])
Anti-TIM-3 monoclonal (TSR-022) +/− nivolumab Advanced solid tumours I NCT02817633 (ref.[169])
Personalized neoantigen-based vaccine + nivolumab, Newly diagnosed, unmethylated glioblastoma I NCT03422094 (ref.[213])
EGFRvIII-specific CAR T cells + anti-PD1 therapy pembrollizumab Newly diagnosed, unmethylated glioblastoma I NCT03726515 (ref.[214])
EGFR806-specific CAR T cells in combination with locoregional immunotherapy (EGFR 806 is a monoclonal antibody recognizing EGFRvIII and a subset of overexpressed wild type EGFRs) EGFR-positive recurrent or refractory paediatric CNS tumours I NCT03638167 (ref.[215])
HER2-specific CAR T cells in combination with locoregional immunotherapy HER2-positive recurrent or refractory paediatric CNS tumours I NCT03500991 (ref.[216])
HER2-specific CAR T cells HER2-positive CNS tumours I NCT02442297 (ref.[217])
IL-13Rα2-specific CAR T cells Recurrent or refractory malignant glioma I NCT02208362 (ref.[218])
CMV pp65 peptide DC vaccine Recurrent medulloblastoma or malignant glioma I NCT03299309 (ref.[219])
CMV pp65 peptide DC vaccine in combination with nivolumab Recurrent brain tumours I NCT02529072 (ref.[220])
IDH1 peptide vaccine Recurrent grade II glioma I NCT02193347 (ref.[221])
Personalized peptide vaccine in combination with TTFields Glioblastoma I NCT03223103 (ref.[222])
Tumour antigen-loaded DC vaccine Brain tumours I NCT03914768 (ref.[223])
IDO inhibitor in combination with temozolomide Primary malignant brain tumours I and II NCT02052648 (ref.[54])
IDO inhibitor (Epacadostat) in combination with nivolumab Select advanced cancers including brain tumours I and II NCT02327078 (ref.[55])
Arginase inhibitor (INCB001158) +/− immune checkpoint blockade therapy Advanced or metastatic solid tumours I and II NCT02903914 (ref.[58])
Autologous CMV-specific CTLs in combination with temozolomide Glioblastoma I and II NCT02661282 (ref.[176])
Nivolumab IDH-mutant gliomas with and without hypermutator phenotype II NCT03718767 (ref.[97])
WT1 peptide vaccine (DSP-7888) in combination with bevacizumab (Anti-VEGFA) Recurrent or progressive glioblastoma following initial therapy II NCT03149003 (ref.[224])
Tumour lysate-pulsed DC vaccine Brain tumours II NCT01204684 (ref.[225])
CMV RNA-loaded DC vaccine Newly diagnosed WHO Grade IV unmethylated glioma II NCT03927222 (ref.[226])
CMV RNA-loaded DC vaccine +/− anti-CD27 therapy varlilumab (to deplete Treg cells but also enhances activation via other mechanisms) Glioblastoma II NCT03688178 (ref.[227])
Temozolomide in combination with radiation +/− nivolumab Newly diagnosed, unmethylated glioblastoma III NCT02667587 (ref.[148])
Nivolumab or temozolomide in combination with radiation Newly diagnosed, unmethylated glioblastoma III NCT02617589 (ref.[147])

CAR, chimeric antigen receptor; CMV, cytomegalovirus; CTLs, cytotoxic T lymphocytes; DC, dendritic cell; EGFRvIII, epidermal growth factor receptor variant III; HER2, human epidermal growth factor receptor 2; IDH, isocitrate dehydrogenase; IDO, indolamine 2,3 -dioxygenase; IL-13Rα2, interleukin 13 receptor α2; LAG3, lymphocyte activation gene 3; PD1, programmed cell death protein 1; TIM3, T cell immunoglobulin and mucin domain-containing protein 3; TTFields, tumour treating fields; WHO, World Health Organization; WT1, Wilms tumour 1.