Dose-ranging efficacy and safety study of topical rapamycin cream
Research type
Research Study
Full title
A Phase 2/3, multi-center, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison of the efficacy and safety of a topical rapamycin cream for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and over
IRAS ID
265239
Contact name
Larissa Kerecuk
Contact email
Sponsor organisation
DSLP
Eudract number
2019-000752-34
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 10 months, 1 days
Research summary
What is the efficacy and safety of different doses of topical rapamycin cream in patients 6-65 years old with facial angiofibroma caused by Tuberous Sclerosis Complex?
Tuberous Sclerosis Complex (TSC) is a disorder characterized by the development of multiorgan benign tumor-like growths in any part of the body. Facial angiofibromas (FA) are present in 70-80% of patients with TSC. Facial angiofibromas in patients with TSC are thought to develop due to the mutational activation of the mTOR pathway. These growths tend to spontaneously bleed or can be affected by slight traumas that can also cause uncontrolled bleeding. When present around the eyes, nose and mouth infections become common. Due to their progressive nature, FA can impair vision and breathing.
Rapamycin is a potent inhibitor of mTOR. Previous clinical studies have found that rapamycin applied topically to FA lesions can reduce the size and redness of the lesions, and in some cases completely clear all FA.
This study will examine the efficacy and safety of two topical rapamycin concentrations (0.5% and 1.0%) over a period of 26 weeks in at least 120 patients. Patients will be required to apply the cream once daily and record this in a diary. Patients will visit the study site seven times during the study, including follow-up. The efficacy of topical rapamycin will be examined using the Investigator’s Global Assessment (IGA) scale, the facial angiofibroma severity index (FASI), as well as subjective and objective improvement ratings. Safety will be assessed with blood tests, urinalysis, vital sign tests and adverse event monitoring up to 4 weeks after the final dose.
The study will be funded by Dermatology Specialties Limited Partnership (DSLP). As TSC is a rare disease, participants will be recruited from multiple sites: in the UK, Australia, New Zealand, Spain and USA.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0324
Date of REC Opinion
14 Nov 2019
REC opinion
Further Information Favourable Opinion