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

Tuberous Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01031901
• Other study ID #: HSC-MS-09-0259
• Status: Completed
• Phase: Phase 1
• First received: December 10, 2009
• Last updated: February 24, 2012
• Start date: December 2009
• Est. completion date: June 2011

Study information

• Verified date: February 2012
• Source: The University of Texas Health Science Center, Houston
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: June 2011
• Est. primary completion date: February 2011
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 13 Years and older

Eligibility

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

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

Related Conditions & MeSH terms

• Angiofibroma
• Nerve Sheath Neoplasms
• Neurofibroma
• Neurofibromatoses
• Sclerosis
• Tuberous Sclerosis

Intervention

Drug:
• Skincerity
Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
• 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
• 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

Locations

Country	Name	City	State
United States	The University of Texas Health Science Center	Houston	Texas

Sponsors (2)

Lead Sponsor	Collaborator
The University of Texas Health Science Center, Houston	Society for Pediatric Dermatology

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rapamycin level		6 months	Yes
Primary	Complete blood count		6 months	Yes
Primary	Total cholesterol		6 months	Yes
Primary	Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)		6 months	Yes
Secondary	Reduction in lesion size and appearance		6 months	No

External Links

•   UT TSC Clinic Home