NCT01031901

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

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: The University of Texas Health Science Center, Houston · Academic / Other
• Sex: All
• Age: 13 Years
• Healthy volunteers: Accepted

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.

Conditions

• Tuberous Sclerosis
• Neurofibromatoses
• Angiofibroma
• Neurofibroma

Interventions

Type	Name	Description
DRUG	Skincerity	Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
DRUG	Skincerity plus sirolimus/rapamycin	Study subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas
DRUG	Skinercity plus sirolimus/rapamycin	Study subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas

Timeline

• Start date: 2009-12-01
• Primary completion: 2011-02-01
• Completion: 2011-06-01
• First posted: 2009-12-15
• Last updated: 2012-02-27

Locations

1 site across 1 country: United States