Texas Children's Cancer and Hematology Center Alpelisib in Pediatric and Adult Patients with Lymphatic Malformations Associated with a PIK3CA Mutation (EPIK-L1)

This Phase 2/3 study evaluates the safety and effectiveness of the targeted medicine Alpelisib in children and adults with lymphatic malformations caused by PIK3CA mutations.

Description

Lymphatic malformations (LyM) are abnormal formations of the lymphatic system that can cause swelling, pain and other complications. This study investigates Alpelisib, a drug that targets the PIK3CA gene mutation often found in patients with LyM. Alpelisib works by blocking a pathway that contributes to the growth of these malformations. This research is important because it offers a targeted treatment option for patients with this specific genetic mutation. The study also includes analysis of biomarkers to better understand treatment response.

Eligibility

• Ages: 0 to 65 years
• Confirmed diagnosis of lymphatic malformation with PIK3CA mutation
• ECOG performance status 0–2
• Adequate organ function
• No prior treatment with Alpelisib

Detailed inclusion and exclusion criteria are listed at clinicaltrials.gov