Alpelisib in PIK3CA-related overgrowth syndrome

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The purpose of this study is to find out if alpelisib (BYL719; the “study drug”), is safe and effective (can help) people who have PIK3CA-related overgrowth spectrum (PROS) disease. 

Eligibility & Criteria

IRB #:
21-018540
Study Phase:
Phase II
Eligible Age Range:
6 - 60 Years
Gender:
All
Study Categories:

Visit Criteria

Participation will last for up to 5 years. Subjects will be assigned to take either the study drug or placebo for the first 16 weeks. From week 17 on, subjects will receive study drug. Study visits include the following assessments: blood draws, ECGs, Echocardiograms, tissue collection (if biopsy is performed as standard of care), whole body MRIs, x-rays, photographs of lesions, lung function tests, walk tests, completion of questionnaires and diaries, and some genetic testing (to confirm mutation status). Reimbursement for travel may be provided.

Leader
Denise Adams

Denise Adams, MD