PIK3CA-Related Overgrowth Spectrum (PROS) (DBCOND0076518)

Identifiers

Synonyms

PIK3CA-related Overgrowth Spectrum / PIK3CA Related Overgrowth Spectrum / Phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha related overgrowth syndrome (disorder)

Associated Data

Indicated Drugs and Targets

Drug	Description	Targets
Alpelisib	Alpelisib is indicated in combination with fulvestrant to treat postmenopausal women, and men, with advanced or metastatic breast cancer. This cancer must be hormone receptor (HR)-positive, human epidermal growth factor...	Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoformtarget Cytochrome P450 3A4enzyme Cytochrome P450 2C9enzyme Cytochrome P450 2C8enzyme Cytochrome P450 2C19enzyme Broad substrate specificity ATP-binding cassette transporter ABCG2transporter ATP-dependent translocase ABCB1transporter

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT05983159	A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations	Alpelisib Mirdametinib	treatment	2	not_yet_recruiting
NCT05563831	National Evaluation of Patients With PIK3CA-Related Overgrowth Spectrum (PROS)	No drug interventions	Not Available	Not Available	recruiting
NCT05294289	Health-related Quality of Life, Symptom Severity, and Pain Among Patients With PIK3CA-related Overgrowth Spectrum: A Mixed-methods Observational Study	No drug interventions	Not Available	Not Available	completed
NCT00001403	Study of Proteus Syndrome and Related Congenital Disorders	No drug interventions	Not Available	Not Available	recruiting
NCT04589650	Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum	Alpelisib	treatment	2	recruiting
NCT04980833	Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)	Alpelisib	treatment	2	active_not_recruiting
NCT04085653	Managed Access Program (MAP) to Provide Access to Alpelisib (BYL719) for Patients With PIK3CA-Related Overgrowth Spectrum (PROS)	Alpelisib	Not Available	Not Available	available
NCT03317366	Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies	Miransertib	Not Available	Not Available	no_longer_available
NCT04285723	Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib	No drug interventions	Not Available	Not Available	completed
NCT02428296	Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation	PIK3CA Sirolimus	treatment	2	completed