Please read below about research opportunities and new targeted treatment medications:

 

Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum

In May, 2019, the FDA approved a PI3K inhibitor manufactured by Novartis Pharmaceuticals for the treatment of breast cancer.  The drug, Piqray (AKA alpelisib and BYL719), is the first FDA-approved PI3K inhibitor on the market specifically targeting patients with a PIK3CA mutation. 

 

Now, Novartis is sponsoring a prospective Phase II multi-center study with an upfront 16-week, randomized, double-blind, placebo-controlled period, and extension periods, to assess the efficacy, safety and pharmacokinetics of alpelisib in eligible pediatric and adult participants with conditions within the PROS (PIK3CA-related overgrowth spectrum) umbrella, including FAVA. Enrollment is currently open in various locations in the United States and Europe.

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Please use this link to learn more:  Alpelisib Study

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Registry for COVID-19 in the Vascular Anomaly Population

If you have a vascular anomaly and contract COVID-19, please ask your doctor to complete the brief questionnaire below. 

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Surveillance Epidemiology of Coronavirus (COVID-19) Under Research Exclusion for Vascular Anomalies

 

Researchers need information on COVID-19 and vascular anomalies. 

 

The SECURE-VA registry is a collaborative worldwide registry for healthcare providers to report COVID-19 in vascular anomaly patients.  We hope to quickly characterize the impact of COVID-19 on people with vascular anomalies. 

 

The study is anonymous and meets exemption criteria under federal regulations. Complete information on security and online collection can be found here. 

 

If you have a vascular anomaly and you contract COVID-19, please provide the link below to your doctor. The form is quick to complete:

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https://www.research.chop.edu/secure-va

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Patient registries provide researchers with the opportunity to analyze anonymous data that is generated and collected over a specific time.  The true analytical value of registries can only be achieved when sufficient numbers of participants provide access to their data.

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