0.1% topical sirolimus in the treatment of cutaneous microcystic lymphatic malformations in children and adults: phase II, split-body randomized, double-blind, vehicle-controlled clinical trial

Phase 1

• Conditions: Cutaneous microcystic lymphatic malformations (CMLM) in children and adults; MedDRA version: 20.0 Level: LLT Classification code 10003229 Term: Arteriovenous malformations System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2018-001359-11-FR
• Lead Sponsor: CHRU TOURS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-07-23
• Last Update Posted: 30 April 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 55

Inclusion Criteria

-Patients = 6 years
-Updated immunization schedule
-Diagnosis of primary cutaneous microcystic lymphatic malformation (CMLM) confirmed by histopathological or dermoscopic examination, with or without an underlying malformation or a syndromic malformation (Protée syndrome for instance), responsible for impairment (oozing, bleeding and/or pain)
-CMLM = 20 cm2, that can be divided into 2 parts of similar severity
-Informed, written consent of the subject and his/her parents if < 18 years
-Rights to French social security (including CMU)
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-Patients with lymphatic malformation requiring a continued backround therapy (involving deep organs)
-Secondary lymphatic malformations (lymphangiectasia post-radiotherapy, etc)
-Previous treatment with oral or topical mTOR inhibitors within 12 months before inclusion
-Previous treatment with oral or topical steroids within 10 days before inclusion
-Immunosuppression (immunosuppressive disease or immunosuppressive treatment)
-Ongoing neoplasia
-Active chronic infectious disease (HBV, HCV, HIV, etc)
-Local fungal, viral (HSV, VZV, etc) or bacterial infection on the site of the CMLM (based on clinical examination)
-Skin necrosis
-Known allergy to one of the components of the topical sirolimus preparation or vehicle
-Women of child-bearing potential (including teenagers) not using a reliable contraceptive method until the end of the study
-Pregnant or breastfeeding women
-Subject already involved in another therapeutic trial

Study & Design

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