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

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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

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

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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