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Alpelisib in PIK3CA-related overgrowth syndrome
This study is now recruiting.
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.
Who Do I Contact?
If you are interested in participating in the study or want to learn more please contact our study team at CVAPResearch [at] chop.edu or 267-426-0743.
Eligibility & Criteria
IRB #:
21-018540
Official Title:
EPIK-P2: A Phase II double-blind study with an upfront, 16- week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)
Study Phase:
Phase II
Eligible Age Range:
6 - 60 Years
Gender:
All
Visit Criteria
Participation will last for up to 5 years. All participants enrolled in January 2025 or later will receive the study drug (no placebo). 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
