TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN
Status:
Recruiting
Trial end date:
2022-09-28
Target enrollment:
Participant gender:
Summary
Lingual microcystic lymphatic malformations (LMLMs) are rare congenital vascular
malformations, presenting as clusters of cysts filled with lymph fluid or blood. They are
responsible for a heavy burden even with small well-limited lesions because of oozing,
bleeding, infections, or even speech, chewing or breathing impairment. Pain and aesthetic
prejudice are also frequently reported. The natural history of LMLMs is progressive
worsening. LMLMs complex management requires multidisciplinary care in specialised centres,
and the "wait-and-see" approach is frequently used. In complicated lymphatic malformations,
whatever the location, treatment with oral sirolimus, an mTOR (mammalian Target of Rapamycin)
inhibitor, is often used.
Topical sirolimus is a known effective treatment for some cutaneous conditions such as
angiofibromas in tuberous sclerosis. Topical applications of sirolimus on the buccal mucosae
have been reported in erosive lichen planus and oral pemphigus vulgaris with good tolerance
and none to slight detectable blood sirolimus concentrations.
The objective of this study is to evaluate the efficacy and safety of a 1mg/mL sirolimus
solution applied once daily on mild to moderate lingual microcystic lymphatic malformation in
children and adults after 4, 8, 12, 16, 20 and 24 weeks of treatment as compared to usual
care (no treatment).