Conditions: Angiofibromas Tuberous Sclerosis
Keywords: Angiofibroma Tuberous Sclerosis Rapamycin Sirolimus mTOR
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 179
Healthy volunteers: no

Brief summary:
The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months.

The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.

Detailed description:

Arms:
arm 1: Cream only arm 2: 0.1% Rapamycin cream arm 3: 1% Rapamycin cream

Interventions: intervention 1: Placebo intervention 2: Rapamycin intervention 3: Rapamycin
Intervention descriptions: intervention 1: Study cream is applied nightly to the affected areas on the face. intervention 2: Study cream is applied nightly to the affected areas on the face. Low Dose intervention 3: Study cream is applied nightly to the affected areas on the face. High Dose

Protocol document: