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

NCT01031901 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 52

Last updated 2012-02-27

No results posted yet for this study

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.

Conditions

  • Tuberous Sclerosis
  • Neurofibromatoses
  • Angiofibroma
  • Neurofibroma

Interventions

DRUG

Skincerity

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

DRUG

Skincerity 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

DRUG

Skinercity 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

Sponsors & Collaborators

  • Society for Pediatric Dermatology

    collaborator OTHER

  • The University of Texas Health Science Center, Houston

    lead OTHER

Principal Investigators

  • Mary Kay Koenig, MD · The University of Texas Health Science Center, Houston

  • Hope Northrup, MD · The University of Texas Health Science Center, Houston

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
13 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2009-12-31
Primary Completion
2011-02-28
Completion
2011-06-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01031901 on ClinicalTrials.gov