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

Sponsor:	The University of Texas Health Science Center, Houston
Collaborator:	Society for Pediatric Dermatology
Information provided by:	The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:	NCT01031901

Purpose

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.

Condition	Intervention	Phase
Tuberous Sclerosis Neurofibromatoses Angiofibroma Neurofibroma	Drug: Skincerity Drug: Skincerity plus sirolimus/rapamycin Drug: Skinercity plus sirolimus/rapamycin	Phase I

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1

Resource links provided by NLM:

Genetics Home Reference related topics: neurofibromatosis type 1 neurofibromatosis type 2 tuberous sclerosis complex Help Me Understand Genetics

MedlinePlus related topics: Neurofibromatosis Tuberous Sclerosis

Drug Information available for: Fluoride Sirolimus Everolimus CCI 779

U.S. FDA Resources

Further study details as provided by The University of Texas Health Science Center, Houston:

Primary Outcome Measures:

Rapamycin level [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Complete blood count [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Total cholesterol [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis) [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Reduction in lesion size and appearance [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment:	60
Study Start Date:	December 2009
Estimated Primary Completion Date:	June 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Placebo Comparator: TSC Placebo Arm TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas	Drug: Skincerity Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
Experimental: TSC 1% Arm TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas	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
Experimental: TSC 5% Arm TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas	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
Placebo Comparator: NF1 Placebo Arm NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas	Drug: Skincerity Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas
Experimental: NF1 1% Arm NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to 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
Experimental: NF1 5% Arm NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to 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

Eligibility

Ages Eligible for Study:	13 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	Yes

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01031901

Locations

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

Sponsors and Collaborators

The University of Texas Health Science Center, Houston

Society for Pediatric Dermatology

Investigators

Principal Investigator:	Mary Kay Koenig, MD	The University of Texas Health Science Center, Houston
Principal Investigator:	Hope Northrup, MD	The University of Texas Health Science Center, Houston

More Information

Additional Information:

UT TSC Clinic Home This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Mary Kay Koenig, MD, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:	NCT01031901 History of Changes
Other Study ID Numbers:	HSC-MS-09-0259
Study First Received:	December 10, 2009
Last Updated:	February 9, 2011
Health Authority:	United States: Institutional Review Board

Keywords provided by The University of Texas Health Science Center, Houston:

Tuberous Sclerosis
Neurofibromatoses
Angiofibroma
Neurofibroma
Sirolimus

Additional relevant MeSH terms:

Neurofibroma
Neurofibromatosis 1
Sclerosis
Tuberous Sclerosis
Neurofibromatoses
Angiofibroma
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases

Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Pathologic Processes
Hamartoma
Malformations of Cortical Development
Nervous System Malformations
Congenital Abnormalities
Neoplasms, Vascular Tissue
Sirolimus
Everolimus
Antibiotics, Antineoplastic
Antineoplastic Agents

ClinicalTrials.gov processed this record on February 09, 2012