Clinical Trial: Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1
Brief Summary:
This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1). Subjects will apply either a Polyvinylidene fluoride (PVDF) coating (Skincerity) containing rapamycin or the PVDF coating alone nightly to fibromatous lesions for a duration of six months.
The primary goal of this study is to evaluate the safety of the topical product in patients with TSC and NF1. The secondary goal of this study is to evaluate the effectiveness of the topical product for treatment of cutaneous fibromatous lesions.
Detailed Summary:
Sponsor: The University of Texas Health Science Center, Houston
Current Primary Outcome:
- Rapamycin level [ Time Frame: 6 months ]
- Complete blood count [ Time Frame: 6 months ]
- Total cholesterol [ Time Frame: 6 months ]
- Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis) [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Reduction in lesion size and appearance [ Time Frame: 6 months ]
Original Secondary Outcome: Same as current
Information By: The University of Texas Health Science Center, Houston
Dates:
Date Received: December 10, 2009
Date Started: December 2009
Date Completion:
Last Updated: February 24, 2012
Last Verified: February 2012
