Alpelisib in PIK3CA Associated Lymphatic Malformations
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Description
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
Eligibility and criteria
What to expect
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.
Related specialties
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