NCT01526356.
Methods | Multicentre prospective, randomised, double‐blind, placebo‐controlled study. |
Participants | 177 participants diagnosed with TSC and visible facial angiofibromas. |
Interventions | Participants were randomised into either: 1. topical placebo cream; OR 2. topical 0.1% rapamycin cream; OR 3. topical 1% rapamycin cream. Study cream is applied nightly to the affected areas on the face. |
Outcomes | Primary outcome: reduction in lesion size and appearance. Photographic, visual, and measurable reduction in the size and severity of the lesions. Secondary outcomes: systemic uptake of topically applied rapamycin; dermatologic sensitivity at the site of application. |
Notes | Sponsored by The University of Texas Health Science Center, Houston. |
IQ: intelligence quotient TSC: tuberous sclerosis complex