Texas Children's Cancer and Hematology Center Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (EPIK-P2) 

Diseases

This is 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 pediatric and adult participants with PIK3CA-related overgrowth spectrum (PROS).

Description

Subjects will be identified from the population of patients diagnosed with PIK3CA-related Overgrowth Spectrum (PROS). Up to ten (10) patients will be enrolled overall.

Eligibility Criteria

• Participants will be enrolled into two age groups: Group 1: ≥ 18 years old and Group 2: 6-17 years old.
• All Sexes are Eligible

Detailed inclusion and exclusion criteria as listed on clinicaltrials.gov