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

The University of Texas System (UT)

Status and phase: Completed; Phase 1

Conditions: Neurofibroma; Neurofibromatoses; Tuberous Sclerosis; Angiofibroma

Treatments: Drug: Skincerity plus sirolimus/rapamycin; Drug: Skincerity; Drug: Skinercity plus sirolimus/rapamycin

Study type: Interventional

Funder types: Other

Identifiers

NCT01031901

HSC-MS-09-0259

Details and patient eligibility

About

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.

Enrollment: 52 patients

Sex: All

Ages: 13+ years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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52 participants in 6 patient groups, including a placebo group

TSC Placebo Arm

Placebo Comparator group

Description:

TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas

Treatment:

Drug: Skincerity

TSC 1% Arm

Experimental group

Description:

TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas

Treatment:

Drug: Skincerity plus sirolimus/rapamycin

TSC 5% Arm

Experimental group

Description:

TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas

Treatment:

Drug: Skinercity plus sirolimus/rapamycin

NF1 Placebo Arm

Placebo Comparator group

Description:

NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas

Treatment:

Drug: Skincerity

NF1 1% Arm

Experimental group

Description:

NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas

Treatment:

Drug: Skincerity plus sirolimus/rapamycin

NF1 5% Arm

Experimental group

Description:

NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas

Treatment:

Drug: Skinercity plus sirolimus/rapamycin