Table 2.

Clinical trials for NF1 associated gliomas.

Drug	Target	Tumor	Phase	Age	Endpoints	Status
Vinblastine +/− Bevacizumab NCT02840409	Cytotoxic/VEGF	LGG	II	6 months to 18 years	Response rate, OS, PFS, visual outcome measures, OCT	Recruiting
Pegylated interferon NCT02343224	Tumor microenvironment	PA or OPG	II	3 to 18 years	Response rate	Recruiting
Pomalidomide NCT02415153	Angiogenesis/immunomodulation	NF1-associated CNS tumors	I	3 to 20 years	Toxicity, MTD	Active, not recruiting
Lenalidomide NCT01553149	Angiogenesis/immunomodulation	PA or OPG	II	0 to 21 years	Response rate	Active, not recruiting
Everolimus (RAD0001) NCT01158651	mTOR	LGG	II	1 to 21 years	Response rate	Active, not recruiting
Binimetinib (MEK162) NCT02285439	MEK	LGG	I/II	1 to 18 years	MTD, response rate	Recruiting
Binimetinib (MEK162) NCT01885195	MEK	Solid tumors with NF1 mutation	II	Older than 18 years	Response rate	Completed (pending results)
Selumetinib NCT01089101	MEK	LGG	I/II	3 to 21 years	Safety, MTD, Response rate	Recruiting
Selumetinib (Selumetinib vs. carboplatin and vincristine) Randomized NCT03871257	MEK	OPG	III	2 to 21 years	Event-free survival ∗, visual acuity	Not yet recruiting
TAK-580 NCT03429803	RAF (pan-RAF kinase inhibitor)	LGG	I/II	1 to 18 years	Toxicity, MTD, 6-month PFS	Recruiting

Abbreviations: LGG, Low-Grade Glioma; MEK, mitogen activate protein kinase; MTD, maximal tolerated dose; mTOR, mammalian target of rapamycin; OPG, Optic-Pathway Glioma; OS, overall survival; PA, Pilocytic Astrocytoma; PFS, progression free survival; RAF, Rapidly accelerated fibrosarcoma; VEGF, vascular endothelial growth factor. ∗ Event-free survival is the time frame from randomization to the first occurrence of any of the following events: clinical or radiographic disease progression, disease recurrence, second malignant neoplasm, or death from any cause, assessed up to 10 years.