Topical Everolimus versus placebo for the treatment of facial angiofibromas in patients with tuberous sclerosis complex.

Phase 1

• Conditions: angiofibromas; MedDRA version: 20.0 Level: PT Classification code 10002429 Term: Angiofibroma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2018-002531-18-FR
• Lead Sponsor: Hospices Civils de Lyon

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-07-11
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 146

Inclusion Criteria

?Male and female subjects > 2 years old.
?Subjects meeting the criteria for a definite or possible diagnosis of TSC
?Subjects with at least 3 FA, diagnosed by a dermatologist
?Women of child-bearing potential with a negative blood pregnancy test at the inclusion.
?Subjects or their parents or legal guardians must provide written informed consent prior to participation in the study
?Subjects covered by French national health insurance

Are the trial subjects under 18? yes
Number of subjects for this age range: 146
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

?Systemic treatment by sirolimus, everolimus, or any other immunosuppressive drug, within 6 months pre inclusion.
?Use of topical tacrolimus or sirolimus on the face, within 6 months pre inclusion.
?Destructive treatment (laser therapy, surgery, cryotherapy) of facial angiofibroma, within 6 months pre inclusion.
?Current concomitant use of topical treatments that could affect facial erythema (e.g. bromonidine)
?Known internal organ involvement requiring systemic mTOR inhibitor in the next 6 months
?Immunosuppression (immunosuppressive disease or immunosuppressive treatment)
?Known chronic infectious disease
?Known hypersensitivity to mTOR inhibitor
?Neutropenia < 1000/mm3
?Thrombopenia < 75,000/mm3
?Chronic renal insufficiency (estimated Glomerular Filtration Rate < 60 mls/min)
?Chronic liver disease (SGOT or SGPT > 3 times upper normal limit)
?Uncontrolled dyslipidaemia
?Uncontrolled diabetes
?Breast feeding or pregnant women, or women of childbearing potential without any effective method of contraception during treatment and up to 12 weeks after treatment discontinuation.
?Subjects who, in the Investigator’s opinion, are unable or unwilling to comply with the protocol.

Study & Design

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