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Table 2 Select ongoing clinical trials of PIK3CA-related disorders using systemic therapies

From: A review of mechanisms of disease across PIK3CA-related disorders with vascular manifestations

Therapy Study name (if applicable) Clinical trial identifier Phase or study type Estimated number of patients Patient population Confirmed PIK3CA mutation required
Sirolimus NCT04598204 2/3 30 1 month to 14 years old with Kaposiform Hemangioendotheliomas, Tufted Angioma, or complicated vascular malformation No
NCT02638389
EudraCT: 2015-001703-32
3 250 3 months to 70 years old with complex vascular anomalies No
NCT03767660 4 20 BRBNS, VMCM, sporadic multiple VM, or large single VM No
Miransertib NCT03317366 Expanded access Not available 2 years and older with overgrowth diseases and/or vascular anomalies Yes (PIK3CA or AKT)
MOSAIC NCT03094832 1/2 85 2 years and older with PROS or PS Yes (PIK3CA or AKT1)
Alpelisib NCT04085653 Managed access Not available 2 years and older with PROS Yes
EPIK-P1 NCT04285723 Chart review 65 2 years and older with PROS Yes
EPIK-P2 NCT04589650 2 150 2 years and older with PROS Yes
  1. BRBNS: Blue Rubber Bleb Nevus Syndrome; PROS: PIK3CA-related Overgrowth Spectrum; PS: Proteus syndrome; VM: venous malformation; VMCM: venous malformation cutaneo-mucosal