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| Name | Class |
|---|---|
| Society for Pediatric Dermatology | OTHER |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TSC Placebo Arm | Placebo Comparator | TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas |
|
| TSC 1% Arm | Experimental | TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas |
|
| TSC 5% Arm | Experimental | TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas |
|
| NF1 Placebo Arm | Placebo Comparator | NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas |
|
| NF1 1% Arm | Experimental | NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Skincerity | Drug | Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas |
|
| Measure | Description | Time Frame |
|---|---|---|
| Rapamycin level | 6 months | |
| Complete blood count | 6 months | |
| Total cholesterol | 6 months | |
| Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis) | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Reduction in lesion size and appearance | 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| 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 | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The University of Texas Health Science Center | Houston | Texas | 77030 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22934754 | Derived | 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. |
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| NF1 5% Arm | Experimental | NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas |
|
| Skincerity plus sirolimus/rapamycin | Drug | 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 | Drug | Study subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to either facial angiofibromas or cutaneous neurofibromas |
|
| ID | Term |
|---|---|
| D014402 | Tuberous Sclerosis |
| D017253 | Neurofibromatoses |
| D018322 | Angiofibroma |
| D009455 | Neurofibroma |
| ID | Term |
|---|---|
| D006222 | Hamartoma |
| D009369 | Neoplasms |
| D009378 | Neoplasms, Multiple Primary |
| D009386 | Neoplastic Syndromes, Hereditary |
| D065703 | Malformations of Cortical Development, Group I |
| D054220 | Malformations of Cortical Development |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009383 | Neoplasms, Vascular Tissue |
| D010524 | Peripheral Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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