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)
Description
This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS).
Eligibility
- Ages from birth to 100 years
- Diagnosis of PROS with symptomatic and/or progressive disease
- At least one measurable lesion
- ECOG performance status 0-2
Detailed inclusion and exclusion criteria are listed on clinicaltrials.gov.
