TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN

Who is this study for? Children, adolescents and adult patients with microcystic lymphatic malformation
What treatments are being studied? Sirolimus Oral Liquid
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Healthy Volunteers: f
View:

• Participants ≥ 5 years of age

• Lingual microcystic lymphatic malformation that does not require systemic treatment, assessed by clinical examination and head-and-neck MRI imaging prior to study enrolment, with or without underlying syndromic malformation (CLAPO for instance)

• Participants covered by or having the rights to social security

• Written informed consent obtained from participant and participant's legal representative if participant is under 18

• Ability for participant to comply with the requirements of the study

Locations
Other Locations
France
REGIONAL Hospital of ORLEANS -Service de Dermatologie
WITHDRAWN
Orléans
Hospital NECKER -AP-HP - Dermatology
ACTIVE_NOT_RECRUITING
Paris
Univsersity of TOURS _ Service de Dermatologie
RECRUITING
Tours
Contact Information
Primary
Annabel MARUANI, MD-PhD
annabel.maruani@univ-tours.fr
+33 247479076
Backup
Wiebe de JONG, MSc
w.dejong@chu-tours.fr
+33 247474680
Time Frame
Start Date: 2020-02-14
Estimated Completion Date: 2025-07-31
Participants
Target number of participants: 12
Treatments
Experimental: Sirolimus 1mg/mL
Application of 1 mg/mL sirolimus solution, 0.5 mL to 1 mL according to the size of the lesion, once daily, on lingual microcystic lymphatic malformation, the experimental intervention versus usual care (no treatment), the control condition.
No_intervention: Control condition
Usual care, i.e. no intervention
Sponsors
Leads: University Hospital, Tours

This content was sourced from clinicaltrials.gov