Topical Everolimus in Patients With Tuberous Sclerosis Complex (EVEROST)
Facial Angiofibromas
About this trial
This is an interventional treatment trial for Facial Angiofibromas focused on measuring Tuberous sclerosis complex (TSC), topical everolimus, seamless design, dose-escalation study, m-TOR inhibitor
Eligibility Criteria
Inclusion Criteria:
- Patients over the age of 2 years
- With definite or possible diagnosis of TSC
- With at least 3 FA, diagnosed by a dermatologist
- Patients (or parents or legal guardians) who have provided written informed consent prior to participation in the study
- Willing and able to comply with study requirements
- With negative blood pregnancy test at the screening visit and using effective contraceptive methods for women of childbearing potential, up to 12 weeks after treatment discontinuation
- Covered by national health insurance
Exclusion Criteria:
- Systemic treatment by sirolimus, everolimus, or any other immunosuppressive drug, during the previous 6 months
- Use of topical tacrolimus or sirolimus on the face, during the previous 6 months
- Destructive treatment (laser therapy, surgery, cryotherapy) of facial angiofibromas during the previous 6 months
- Concomitant use of topical treatments that could affect facial erythema (e.g. Brimonidine)
- 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
- Brest feeding or pregnant women, or women on childbearing age 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.
Sites / Locations
- Hopital Femme Mère Enfant
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Experimental
Experimental
Placebo Comparator
Topical everolimus 0.1%
Topical everolimus 0.5%
Topical everolimus 1%
Topical placebo
Everolimus topical formulation will be applied to the affected areas, once daily, in the evening, for 6 months. Dose regimens will be identical, regardless of the dose-strength of the topical formulation. The topical formulation will be applied onto areas of the face affected by FA by the patient or the patient's parents/legal guardians, while observing standardised precautions (avoiding the eyes, washing the hands after application, etc.).
Everolimus topical formulation will be applied to the affected areas, once daily, in the evening, for 6 months. Dose regimens will be identical, regardless of the dose-strength of the topical formulation. The topical formulation will be applied onto areas of the face affected by FA by the patient or the patient's parents/legal guardians, while observing standardised precautions (avoiding the eyes, washing the hands after application, etc.).
Everolimus topical formulation will be applied to the affected areas, once daily, in the evening, for 6 months. Dose regimens will be identical, regardless of the dose-strength of the topical formulation. The topical formulation will be applied onto areas of the face affected by FA by the patient or the patient's parents/legal guardians, while observing standardised precautions (avoiding the eyes, washing the hands after application, etc.).
Topical placebo will be identical to the everolimus topical formulation. Topical placebo will be applied to the affected areas, once daily, in the evening, for 6 months by the patient or the patient's parents/legal guardians, while observing standardised precautions (avoiding the eyes, washing the hands after application, etc.).