Table 2.

Summary of recent clinical trials of targeted therapies for pediatric glioma.

Author	Year	ClinicalTrials.gov ID	Phase	Patients	Disease	Molecular Target	Treatment	Outcome
LGG
Fangusaro et al. [68]	2019	NCT01089101	II	3–21 y n = 38	Recurrent/refractory LGG	MEK	Selumetinib	Positive antitumor activity Well-tolerated
Hargrave et al. [62]	2019	NCT01677741	I/II	2–18 y n = 32	Recurrent/refractory LGG	BRAF V600E mutant	Dabrafenib	Positive antitumor activity Well-tolerated
Nicolaides et al. [70]	2020	NCT01748149	I	3–17 y n = 19	Recurrent/refractory gliomas	BRAF V600E mutant	Vemurafenib	Positive antitumor activity Well-tolerated
HGG
Wetmore et al. [73]	2016	NCT01462695	II	18 m–22 y n = 30	HGG or ependymoma	VEGFR PDGFR KIT	Sunitinib	No antitumor activity Well-tolerated
Becher et al. [74]	2017	NCT01049841	I	4–24 y n = 23	Recurrent/refractory brain tumor	AKT mTOR	Perifosine Temsirolimus	Well-tolerated
Broniscer et al. [75]	2018	NCT01644773	I	2–21 y n = 25	Recurrent/progressive HGG or DIPG	PDGFRA c-Met	Dasatinib Crizotinib	Minimal antitumor activity Poorly tolerated
Chi et al. [76]	2019	NCT03134131	II	3–42 y n = 18	H3K27M mutant diffuse midline glioma/DIPG	DRD2/3	ONC201	Positive antitumor activity

LGG, low-grade glioma; y, years; HGG, high-grade glioma; m, months; DIPG, diffuse intrinsic pontine glioma; DRD2/3, dopamine receptor type 2/3.