Status:
COMPLETED
TOPical Sirolimus in linGUal Microcystic Lymphatic Malformation -TOPGUN
Lead Sponsor:
University Hospital, Tours
Conditions:
Lingual Microcystic Lymphatic Malformations
Eligibility:
All Genders
5+ years
Phase:
PHASE2
Brief 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 burde...
Detailed Description
This is a randomized, open-labelled, multicenter pilot study using an individually randomized stepped wedge design over a 24 weeks period to evaluate topical application of 1 mg/mL sirolimus solution,...
Eligibility Criteria
Inclusion
- 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
Exclusion
- Patients with a lymphatic malformation requiring a continued background therapy (involving deep organs)
- Secondary lymphatic malformations (lymphangiectasia post-radiotherapy, etc)
- Previous treatment with systemic or topical mTOR (mammilian target of rapamycin) inhibitors within 12 months before inclusion (half-life of oral sirolimus is 60 days in adults).
- Previous treatment with oral or topical steroids within 10 days before inclusion (half-life of corticosteroids is 12-36 hours)
- Immunosuppression (immunosuppressive disease or immunosuppressive treatment)
- Ongoing neoplasia
- Active chronic infectious disease (Hepatitis-B virus, Hepatitis-C virus, HIV, etc)
- Local necrosis
- Local fungal, viral (herpes simplex virus, varicella zoster virus, etc) or bacterial infection on the site of the LMLM (based on clinical examination)
- Known allergy to one of the components of the sirolimus solution
- Soy bean or Peanut allergy
- Pregnant or breastfeeding women
- Women of child-bearing potential (including teenagers) not using a reliable contraceptive method until the end of the study and three month after the end of the study or sirolimus discontinuation.
- Already involved in another therapeutic trial
Key Trial Info
Start Date :
February 14 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 8 2025
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT04128722
Start Date
February 14 2020
End Date
January 8 2025
Last Update
November 24 2025
Active Locations (2)
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1
Univsersity of TOURS _ Service de Dermatologie
Tours, Indre et Loire, France, 37044
2
Hospital NECKER -AP-HP - Dermatology
Paris, France, 75015