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Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

2014-08-27 03:17:18 | BioPortfolio

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.

Study Design

Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Conditions

Tuberous Sclerosis

Intervention

Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin

Location

The University of Texas Health Science Center
Houston
Texas
United States
77030

Status

Recruiting

Source

The University of Texas Health Science Center, Houston

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:17:18-0400

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