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NCT Number: NCT01031901

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

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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Key information

Conditions

Tuberous Sclerosis Angiofibroma Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hamartoma Heredodegenerative Disorders, Nervous System Malformations of Cortical Development Malformations of Cortical Development, Group I Neoplasms Neoplasms by Histologic Type Neoplasms, Multiple Primary Neoplasms, Nerve Tissue Neoplasms, Vascular Tissue Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Nervous System Malformations Nervous System Neoplasms Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibromatoses Neuromuscular Diseases Peripheral Nervous System Diseases Peripheral Nervous System Neoplasms

Age range

13 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

The University of Texas Health Science Center

Houston, Texas, 77030, United States

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Skincerity

Drug

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

Primary outcomes

  1. Rapamycin level

    Time frame: 6 months

  2. Complete blood count

    Time frame: 6 months

  3. Total cholesterol

    Time frame: 6 months

  4. Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)

    Time frame: 6 months

Secondary outcomes

  1. Reduction in lesion size and appearance

    Time frame: 6 months

Sponsors and collaborators

Lead sponsor

The University of Texas Health Science Center, Houston

Other

Collaborators

  • Society for Pediatric Dermatology

Registry information

Official study title

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

Important dates

Study start
2009
Primary completion
2011
Study completion
2011
First posted
Dec 15, 2009
Registry last updated
Feb 27, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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