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

This study has been completed.
Sponsor:
Collaborator:
Society for Pediatric Dermatology
Information provided by (Responsible Party):
Mary Kay Koenig, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:
NCT01031901
First received: December 10, 2009
Last updated: February 24, 2012
Last verified: February 2012
History of Changes
  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: 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:

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: 52
Study Start Date: December 2009
Study Completion Date: June 2011
Primary Completion Date: February 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 by The University of Texas Health Science Center, Houston

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Mary Kay Koenig, Assistant Professor, Pediatrics-Neurology, 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 24, 2012
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 May 19, 2013