TOPical sirolimus in linGUal microkystic lymphatic malformation-TOPG

Phase 1

• Conditions: ingual microcystic lymphatic malformations (LMLM) in children and adults; MedDRA version: 20.0Level: LLTClassification code 10003229Term: Arteriovenous malformationsSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2019-001530-33-FR
• Lead Sponsor: CHRU TOURS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-04-01
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

-Patients = 5 years
-Lingual microcystic lymphatic malformation Wiegand stage I or IIa (Wiegand 2009) 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

Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-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 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 (HBV, HCV, HIV, etc)
-Local necrosis
-Local fungal, viral (HSV, VZV, 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
-Already involved in another therapeutic trial

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified