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National Institutes of Health

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC)
and Neurofibromatosis I (NF1)
ClinicalTrials.gov Identifier:

This study has been completed.

NCT01031901

Sponsor:

First received: December 10, 2009


Last updated: February 24, 2012
Last verified: February 2012
History of Changes

The University of Texas Health Science Center, Houston


Collaborator:

Society for Pediatric Dermatology


Information provided by (Responsible Party):

Mary Kay Koenig, The University of Texas Health Science Center, Houston
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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 1

Study Type:
Study Design:

Interventional
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
MedlinePlus related topics: Neurofibromatosis Tuberous Sclerosis
Drug Information available for: Sirolimus Everolimus Temsirolimus
Genetic and Rare Diseases Information Center resources: Bourneville Syndrome Nerve Sheath Neoplasm Neurofibroma Neurofibromatosis
Neurofibromatosis Type 1 Tuberous Sclerosis
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 ]
Enrollment:
Study Start Date:
Study Completion Date:
Primary Completion Date:

52
December 2009
June 2011
February 2011 (Final data collection date for primary outcome measure)

Arms

Assigned Interventions

Placebo Comparator: TSC Placebo Arm

Drug: Skincerity

TSC subjects will apply a study product containing


polyvinylidene fluoride coating alone to facial angiofibromas

Study subjects will apply a study product containing polyvinylidene fluoride


coating alone to either facial angiofibromas or cutaneous neurofibromas

Experimental: TSC 1% Arm

Drug: Skincerity plus sirolimus/rapamycin

TSC subjects will apply a study product containing


polyvinylidene fluoride coating plus 1 mg of
sirolimus/rapamycin to facial angiofibromas

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

Drug: Skinercity plus sirolimus/rapamycin

TSC subjects will apply a study product containing


polyvinylidene fluoride coating plus 5 mg of
sirolimus/rapamycin to facial angiofibromas

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

Drug: Skincerity

NF1 subjects will apply a study product containing


polyvinylidene fluoride coating alone to cutaneous
neurofibromas

Study subjects will apply a study product containing polyvinylidene fluoride


coating alone to either facial angiofibromas or cutaneous neurofibromas

Experimental: NF1 1% Arm

Drug: Skincerity plus sirolimus/rapamycin

NF1 subjects will apply a study product containing


polyvinylidene fluoride coating plus 1 mg of
sirolimus/rapamycin to cutaneous neurofibromas

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

Drug: Skinercity plus sirolimus/rapamycin

NF1 subjects will apply a study product containing


polyvinylidene fluoride coating plus 5 mg of
sirolimus/rapamycin to cutaneous neurofibromas

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:
Genders Eligible for Study:
Accepts Healthy Volunteers:

13 Years and older


Both
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


Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a
study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general
information, see Learn About Clinical Studies.
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
No publications provided by The University of Texas Health Science Center, Houston
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Koenig MK, Hebert AA, Roberson J, Samuels J, Slopis J, Woerner A, Northrup H. Topical rapamycin therapy to alleviate the cutaneous
manifestations of tuberous sclerosis complex: a double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied
rapamycin. Drugs R D. 2012 Sep 1;12(3):121-6. doi: 10.2165/11634580-000000000-00000.
Responsible Party:
ClinicalTrials.gov Identifier:
Other Study ID Numbers:
Study First Received:
Last Updated:
Health Authority:

Mary Kay Koenig, Assistant Professor, Pediatrics-Neurology, The University of Texas Health Science Center, Houston
NCT01031901 History of Changes
HSC-MS-09-0259
December 10, 2009
February 24, 2012
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
Neurofibromatoses

Nerve Sheath Neoplasms


Nervous System Diseases

Neurofibromatosis 1
Sclerosis
Tuberous Sclerosis
Congenital Abnormalities
Genetic Diseases, Inborn
Hamartoma
Heredodegenerative Disorders, Nervous System
Malformations of Cortical Development
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Multiple Primary
Neoplasms, Nerve Tissue
Neoplastic Syndromes, Hereditary
ClinicalTrials.gov processed this record on March 31, 2015

Nervous System Malformations


Nervous System Neoplasms
Neurocutaneous Syndromes
Neurodegenerative Diseases
Neuromuscular Diseases
Pathologic Processes
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms
Everolimus
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic