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Topical Sirolimus for Microcystic Lymphatic Malformations
This study is now recruiting.
The purpose of this study is to see if a new topical (applied to the skin) formulation of rapamycin, PTX-022, is safe and effective in treating participants with microcystic Lymphatic Malformations (mLM).
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 #:
21-019452
Official Title:
A Multicenter, Phase 2, Open-label Study Evaluating the Safety and Efficacy of Sirolimus 3.9% Topical Gel (PTX-022) in the Treatment of Microcystic Lymphatic Malformations
Study Phase:
Phase II
Eligible Age Range:
13 - 40 Years
Gender:
All
Visit Criteria
The purpose of this study is to evaluate the safety and tolerability of the study drug PTX-022. The study will enroll male and female patients between 13 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 about 20 weeks and 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 expenses and compensation for participation may be provided.
Leaders
