Table 1.

Clinical trials dedicated to vascular malformations.

Vascular malformation types	Target	Drug	Main objective	Phase	Clinical trial ID number
Lymphatic Malformations	mTOR	Sirolimus	Safety and efficacy	2–3	NCT06673290
Microcystic Lymphatic Malformations	mTOR	Sirolimus	Safety and efficacy	Not applicable	NCT06160739
Cervico-facial Lymphatic Malformations	mTOR	Sirolimus	Safety and efficacy	2	NCT03243019
Microcystic Lymphatic Malformations	mTOR	Topical sirolimus	Safety and efficacy	2	NCT04128722
Lymphatic Malformations	PI3Kα	Alpelisib	Safety and efficacy	2–3	NCT05948943
Brainstem Cavernous Malformations	mTOR	Sirolimus	Safety and efficacy	2	NCT06091332
Slow-Flow Vascular Malformations	PI3Kα	Alpelisib	Safety and efficacy	2	NCT05983159
MCAP	PI3Kα	Alpelisib	Safety and efficacy	2	NCT05577754
Arteriovenous Malformations	mTOR	Sirolimus	Safety and efficacy	2	NCT02042326
Arteriovenous Malformations	MEK	Trametinib	Safety and efficacy	2	NCT06098872
Arteriovenous Malformations	MEK	Trametinib	Safety and efficacy	2	NCT04258046
Arteriovenous Malformations	MEK	Trametinib	Safety and efficacy	2	EudraCT 2019-003573-26
Extracranial Arteriovenous Malformations	MEK	Cobimetinib	Safety and efficacy	2	NCT05125471
Fast-Flow Vascular Malformations	MEK	Mirdametinib	Safety and efficacy	2	NCT05983159
Hereditary Hemorrhagic Telangiectasia	AKT	VAD044	Safety and efficacy	1	NCT05406362
Hereditary Hemorrhagic Telangiectasia	VEGFR	Pazopanib	Efficacy	1–2	NCT03850730
Hereditary Hemorrhagic Telangiectasia	VEGFR	Pazopanib	Safety and efficacy	2–3	NCT03850964

MCAP megalencephaly-capillary malformation polymicrogyria syndrome.