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Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC)
and Neurofibromatosis I (NF1)
ClinicalTrials.gov Identifier:
NCT01031901
Sponsor:
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
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:
52
December 2009
June 2011
February 2011 (Final data collection date for primary outcome measure)
Arms
Assigned Interventions
Drug: Skincerity
Drug: Skincerity
Eligibility
Ages Eligible for Study:
Genders Eligible for Study:
Accepts Healthy Volunteers:
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
Locations
United States, Texas
Principal Investigator:
Principal Investigator:
Hope Northrup, MD
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
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