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. While the study is active, enrollment is currently closed.

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Please use this link to learn more:  Alpelisib Study

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ReInspire Study

Relay Therapeutics’ clinical trial for individuals with vascular malformations, including FAVA, is currently enrolling eligible individuals 6 years and older.  

 

The clinical trial is evaluating the investigational drug RLY-2608 (zovegalisib), to learn more about the safety and effectiveness of RLY-2608 in the treatment of individuals with PIK3CA-driven vascular malformations, including FAVA.  

 

You can learn more about the trial here: https://clinicaltrials.gov/study/NCT06789913 (global audience) or https://reinspirestudy.com/  (US-audience)

 

Questions for Relay Therapeutics? Email clinicaltrials@relaytx.com  

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