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

Phase 1

Completed

• Conditions: Tuberous Sclerosis; Neurofibromatoses; Angiofibroma; Neurofibroma
• Interventions: Drug: Skincerity; Drug: Skincerity plus sirolimus/rapamycin; Drug: Skinercity plus sirolimus/rapamycin

• Registration Number: NCT01031901
• Lead Sponsor: The University of Texas Health Science Center, Houston

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 Description

Not available

Study Timeline

• Study Start: 2009-12
• Study First Submitted: 2009-12-10
• Study First Submitted QC: 2009-12-14
• Study First Posted: 2009-12-15
• Primary Completion: 2011-02
• Study Completion: 2011-06
• Status Verified: 2012-02
• Last Update Submitted: 2012-02-24
• Last Update Posted: 2012-02-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 52

Inclusion Criteria

• Subject is willing and able to comply with all trial requirements
• Subject is male or female and over 13 years of age
• Subject has a diagnosis of either TSC or NF1 and has visible fibromatous lesions (angiofibromas or neurofibromas)
• Female subjects of child-bearing potential must not be pregnant and must agree to use appropriate contraceptive methods for the duration of the trial

Exclusion Criteria

• Subject is currently receiving therapy with rapamycin or sirolimus
• Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction
• Subject is currently participating in or has participated within the last 30 days in any clinical trial involving an investigational drug
• Subject has a known hypersensitivity to either the PVDF coating (Skincerity®) or rapamycin
• Subject is a pregnant or nursing female

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
TSC Placebo Arm	Skincerity	TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
TSC 1% Arm	Skincerity plus sirolimus/rapamycin	TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
TSC 5% Arm	Skinercity plus sirolimus/rapamycin	TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
NF1 Placebo Arm	Skincerity	NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
NF1 1% Arm	Skincerity plus sirolimus/rapamycin	NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
NF1 5% Arm	Skinercity plus sirolimus/rapamycin	NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas

Primary Outcome Measures

Name	Time	Method
Rapamycin level	6 months
Complete blood count	6 months
Total cholesterol	6 months
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)	6 months

Secondary Outcome Measures

Name	Time	Method
Reduction in lesion size and appearance	6 months

Trial Locations

Locations (1)

The University of Texas Health Science Center; 🇺🇸 Houston, Texas, United States