NCT01526356

Topical Rapamycin to Erase Angiofibromas in TSC

Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 179 (actual)
• Sponsor: The University of Texas Health Science Center, Houston · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Angiofibromas
• Tuberous Sclerosis

Interventions

Type	Name	Description
DRUG	Placebo	Study cream is applied nightly to the affected areas on the face.
DRUG	Rapamycin	Study cream is applied nightly to the affected areas on the face. Low Dose
DRUG	Rapamycin	Study cream is applied nightly to the affected areas on the face. High Dose

Timeline

• Start date: 2012-05-01
• Primary completion: 2014-08-01
• Completion: 2014-08-01
• First posted: 2012-02-03
• Last updated: 2020-10-26
• Results posted: 2020-10-26

Locations

10 sites across 2 countries: United States, Australia