Topical Everolimus in Patients With Tuberous Sclerosis Complex

• Register: ClinicalTrials.gov
• Last refreshed on: 10 May 2021
• Main ID: NCT02860494
• Date of registration: 04/08/2016
• Prospective Registration: Yes
• Primary sponsor: Hospices Civils de Lyon
• Public title: Topical Everolimus in Patients With Tuberous Sclerosis Complex EVEROST
• Scientific title: Topical Everolimus Versus Placebo for the Treatment of Facial Angiofibromas in Patients With Tuberous Sclerosis Complex. A Phase II/III, Multicentre, Randomized, Double-blind, Placebo-controlled Study of 3 Doses of Topical Everolimus.
• Date of first enrolment: December 2020
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02860494
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

France

Contacts

• Name: Alice PHAN, MD
• Affiliation: Hospices Civils de Lyon

Key inclusion & exclusion 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.



Age minimum: N/A
Age maximum: 2 Years
Gender: All

Health Condition(s) or Problem(s) studied

Facial Angiofibromas

Intervention(s)

Drug: Placebo

Drug: Everolimus

Primary Outcome(s)

Facial Angiofibroma Severity Index (FASI) [Time Frame: 6 months]

Secondary Outcome(s)

Dermatologist's global assessment of efficacy [Time Frame: 6 months]

Patient or parents self-assessment [Time Frame: 6 months]

Dermatological quality of life [Time Frame: 6 months]

scaling scores [Time Frame: 6 months]

blood levels of topically applied everolimus [Time Frame: 6 months]

FA size [Time Frame: 6 months]

dryness score [Time Frame: 6 months]

Local tolerance of the topically applied formulation using patient self-assessment [Time Frame: 6 months]

Secondary ID(s)

2018-002531-18

69HCL16_0062

Source(s) of Monetary Support

Please refer to primary and secondary sponsors