Research and Trials

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

 

 

VACOM:  A study on Communications in Vascular Anomalies

Dr. Bryan Sisk and Dr. Anna Kerr at Washington University in St. Louis are conducting a Vascular Anomalies Communications (VACOM) Study and are seeking participants with FAVA and other vascular anomalies.  This study is open to caregivers, as well as adults ages 18-39, who live in the United States. The total time commitment is one 45-60 minute phone interview, and participants receive a $40 Amazon gift card for their time.  

 

The findings from this research will be essential to develop future interventions to improve medical care and communication for families who experience barriers in dealing with their and their loved one's FAVA and other vascular anomalies.  Contact Dr. Bryan Sisk with any questions at siskb@wustl.edu 

 

Please register today using this link:  VACOM Study

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.

Please use this link to learn more:  Alpelisib Study

Managed Access Program (MAP) to Provide Access to Alpelisib (BYL719) for Patients With PIK3CA-Related Overgrowth Spectrum (PROS)

The Novartis Managed Access Program (MAP) provides access to alpelisib for patients diagnosed with conditions within the PROS (PIK3CA-related overgrowth spectrum) umbrella, including FAVA, who fulfill certain eligibility criteria. Enrollment is currently open in various locations worldwide, including but not limited to the United States, Europe, Australia, Canada, Mexico, India, Israel, and Singapore.   

Please use this link to learn more:  Alpelisib MAP Program 

Patients should contact their treating physicians who can, if clinically appropriate, make a request to Novartis for access to treatment using this link on the Novartis website:  Managed Access Program

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:

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

 

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.

_edited.png

Photo courtesy of  Sippakorn Yamkasikorn on Unsplash