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Topical Sirolimus for Microcystic Lymphatic Malformations

Topical Sirolimus for Microcystic Lymphatic Malformations

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If you are interested in participating in the study or want to learn more, please get in touch. Contact us
This study is now recruiting. Learn more about enrolling here.

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


IRB Number:
21-019452
Eligible age range:
13 years - 40 years
Clinical trial phase:
Phase II
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

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.

Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.

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