This is a study to understand how safe and effective a medication (alpelisib) is in patients with PIK3CA Related Overgrowth Syndromes (PROS). We will be studying adults and children who are at least 6 years of age.
At each visit you (or your child) will meet with members of the study team. Visits for the first year are monthly then every 6 months for the rest of the study. Visits will take anywhere from 2 hrs to 2 days as an outpatient and may include talking, physical examination, questionnaires, blood draws, studies of heart and lung function (EKGs, echocardiograms, lung function tests), MRI scans and photographs of your (or your child's) body, and other X-rays.
Gas and parking assistance, study drug at no cost for the duration of the study, meals for long clinic visits, hotel assistance for 2-day visits.
Pediatrics - Hematology/Oncology
Clinical or Medical