Alpelisib in PIK3CA Associated Lymphatic Malformations



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This study has several parts and involves taking a study drug called alpelisib. Some participants enrolled into Stage 2 will receive a placebo for the first part of the study (24 weeks) and will then receive the study drug. The purpose of this study is to find out if the study drug is safe and effective (can help) people with a confirmed diagnosis of lymphatic malformation (LyM) and a documented mutation in the PIK3CA gene.

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] or 267-426-0743. Identifier

Eligibility & Criteria

IRB #:
Official Title:
A Two-Stage Double-Blind, Randomized, Placebo-Controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients with Lymphatic Malformations Associated with a PIK3CA Mutation
Study Phase:
Phase II
Phase III
Eligible Age Range:
2 - 60 Years
Study Categories:

Visit Criteria

This study has several parts. Only Stage 1 is currently open at this time. The study will enroll male and female patients 2 years of age and older that have been diagnosed with lymphatic malformations associated with a PIK3CA mutation. Participation will last approximately 3 years. In addition to receiving study drug, visits include the following assessments: tissue collection, research biopsy, blood draws, ECGs, MRIs, x-rays, photographs of lesions, completion of questionnaires, interviews, and diaries, and some genetic testing (to confirm mutation status). It is possible that participants may require IV contrast and sedation/anesthesia as part of the research biopsy and/or MRI.

Reimbursement for travel may be provided.