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)

This Phase 2 study evaluates the targeted medicine Alpelisib in children and adults with symptomatic or progressive PIK3CA-Related Overgrowth Spectrum (PROS).

Description

PIK3CA-Related Overgrowth Spectrum (PROS) is a group of rare conditions caused by mutations in the PIK3CA gene, leading to abnormal tissue growth including blood vessels malformations. Alpelisib is a targeted therapy that blocks the activity of the PIK3CA gene, potentially reducing or stabilizing the overgrowth and improving symptoms. This study is important because it explores a treatment option for patients with limited alternatives. It includes both children and adults and aims to assess how well Alpelisib works and how safe it is. The study also includes biomarker analysis to better understand treatment response.

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.