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

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01031901

Enrollment

Registered

2009-12-15

Start date

2009-12-31

Completion date

2011-06-30

Last updated

2012-02-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Angiofibroma, Neurofibroma, Neurofibromatoses, Tuberous Sclerosis

Keywords

Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma, Sirolimus

Brief summary

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.

Interventions

DRUGSkincerity

Study subjects will apply a study product containing polyvinylidene fluoride coating alone to either facial angiofibromas or cutaneous neurofibromas

DRUGSkincerity 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

DRUGSkinercity 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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

13 Years to No maximum

Healthy volunteers

Yes

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

Design outcomes

Primary

Measure	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

Secondary

Measure	Time frame
Reduction in lesion size and appearance	6 months

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 25, 2026