Topical Sirolimus for Microcystic Lymphatic Malformations
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Description
The purpose of this study is to see if a newtopical (applied to the skin) formulation of rapamycin, PTX-022, is safe andeffective in treating participants with microcystic Lymphatic Malformations(mLM).
Eligibility and criteria
What to expect
The purpose of this study is to evaluate thesafety and tolerability of the study drug PTX-022. The study will enroll maleand female patients between 13 and 60 years of age that have been diagnosedwith a microcystic lymphatic malformation with a history of frequent leakingand/or bleeding. Subjects will receive PTX-022, which is a topical gel that willbe applied directly to the lesion. Participation will last for about 20 weeksand will include 8 visits. Study visits include the following assessments:physical exams, vital signs, blood and urine tests, photography of the lesions,and the completion of diaries and questionnaires. Reimbursement for travel expensesand compensation for participation may be provided.
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