Topical Sirolimus for Microcystic Lymphatic Malformations

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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
Study Categories:

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.