PTX-022 in the Treatment of Microcystic Lymphatic Malformations
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Description
The purpose of this study is to see if a new investigational topical (applied to the skin) formulation of sirolimus, PTX-022 (the study drug), is safe and effective in treating participants with microcystic Lymphatic Malformations.
Eligibility and criteria
What to expect
This study will enroll patients between 6 and 60 years of age that have been diagnosed with a microcystic lymphatic malformation with a history of frequent leaking and/or bleeding. Subjects will receive PTX-022, which is a topical gel that will be applied directly to the lesion. Participation will last for at least 14 months and will include 5 in-person visits and at least 3 remote visits (by phone call). Study visits include the following assessments: physical exams, vital signs, safety blood and urine tests, research blood tests, photography of the lesions, completion of interviews, and questionnaires. Reimbursement for travel expenses and compensation for participation may be provided.
Related specialties
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