Topical Everolimus in Patients With Tuberous Sclerosis Complex
Status:
Withdrawn
Trial end date:
2024-12-01
Target enrollment:
Participant gender:
Summary
Tuberous sclerosis complex (TSC) is a rare genetic multisystem disorder characterized by the
development of hamartomas in several organs (e.g. brain, heart, kidney, liver, lung), and
skin in more than 90% of cases. Facial angiofibromas (FA), present in about 80% of patients,
are a stigmatizing hallmark of the disease. Everolimus could be a candidate for use as a
topical formulation to treat FA. This adaptive seamless Phase II/III study primary objective
is to determine the dose of topical everolimus for treatment of FA and evaluate the efficacy
and safety of topical everolimus versus placebo in patients with angiofibromas.