Research and Trials
Please read below about research opportunities and new targeted treatment medications:
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.
Please use this link to learn more: Alpelisib Study
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.
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:
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.