Clinical Trial: Topical Rapamycin to Erase Angiofibromas in TSC

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 Summary:
Sponsor: The University of Texas Health Science Center, Houston

Current Primary Outcome: Reduction in lesion size and appearance [ Time Frame: 6 months ]

Photographic, visual, and measurable reduction in the size and severity of the lesions.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Confirm the lack of systemic uptake of topically applied rapamycin. [ Time Frame: 6 months ]
    Blood levels checked to confirm the lack of systemic rapamycin.
  • Dermatologic sensitivity at the site of application. [ Time Frame: 6 months ]
    Pain, erythema, or pruritis at the application site.


Original Secondary Outcome: Same as current

Information By: The University of Texas Health Science Center, Houston

Dates:
Date Received: February 1, 2012
Date Started: May 2012
Date Completion:
Last Updated: June 9, 2015
Last Verified: June 2015