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 April, 2022, the U.S. FDA granted accelerated approved for a PI3K inhibitor manufactured by Novartis Pharmaceuticals for the treatment of adult and pediatric patients with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy.  The drug, Vijoice (AKA alpelisib and BYL719), is the first FDA-approved treatment of PROS.  In accordance with the Accelerated Approval Program, continued approval is contingent upon further trials.  

 

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 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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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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