Topical Sirolimus in Cutaneous Lymphatic Malformations
Status:
Recruiting
Trial end date:
2023-06-01
Target enrollment:
Participant gender:
Summary
Cutaneous microcystic lymphatic malformations (CMLMs) are rare conditions of children and
adults resulting from abnormal embryologic development of lymphatic vessels. They present as
clusters of vesicles full of lymph and blood of various extent. They ooze and bleed, inducing
maceration, esthetic impairment, scars, pain, bacterial infections and impaired quality of
life. Currently, treatments for CMLMs are disappointing, and their management is challenging.
Sirolimus is an inhibitor of mammalian target of rapamycin (mTOR), a serine/threonine protein
kinase involved in cell growth and proliferation, cellular metabolism, autophagy and
angio-lymphangiogenesis. Topical sirolimus, known to be efficient and well tolerated in
cutaneous angiofibromas linked to tuberous sclerosis, has recently been reported effective in
few reports of patients with CMLMs. The objective of this trial is to compare the efficacy
and safety of a 12-week application of 0.1% topical sirolimus versus topical vehicle in CMLMs
in children and adults.