Status:

COMPLETED

Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

Lead Sponsor:

Le Bonheur Children's Hospital

Collaborating Sponsors:

Pfizer

University of Tennessee

Conditions:

Facial Angiofibroma

Eligibility:

All Genders

3-45 years

Phase:

PHASE1

Brief Summary

The objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial an...

Detailed Description

This is an open label, prospective single center study. Patients that are seen in our regional Tuberous Sclerosis Complex Clinic and have facial angiofibromas that would benefit from this cream and wh...

Eligibility Criteria

Inclusion

  • Patient or his / her legally authorize representative (LAR) must sign and date the approved informed consent prior to study participation or initiation of study procedures. If appropriate, the patient will give written or verbal consent. Surrogate consent will be obtained utilizing the "legally authorized representative (LAR)". The LAR must be an adult who has exhibited special care and concern for the subject, who is familiar with the subject's personal values, who is reasonably available, and who is willing to serve. No person who is identified as a protective order or other court order that directs that person to avoid contact with the subject shall be eligible to serve as the subject's LAR. Identification of LAR should normally be made using the following order of descending preference: Conservator, guardian, attorney in fact, subject's spouse (unless legally separated), the subject's adult child, the subject's parent, the subject's adult sibling, any other adult relative of the subject, or other adult who is familiar with the patient's personal values, who is reasonably available, and who is willing to serve.
  • Patient must have a diagnosis of Tuberous Sclerosis Complex.
  • Female or male patients over the age of 3 years.
  • Female subjects of child bearing potential must not be pregnant and must undergo a pregnancy test, and must agree to use appropriate contraceptive methods

Exclusion

  • Patients will be excluded from entry into the study if any of the following are true:
  • Patient has a history of drug allergy to rapamune.
  • Patient is pregnant or lactating.
  • Subject is receiving therapy with Rapamycin.
  • 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 a clinical trial involving an investigative drug.
  • Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas.
  • Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.

Key Trial Info

Start Date :

October 1 2013

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

July 1 2016

Estimated Enrollment :

11 Patients enrolled

Trial Details

Trial ID

NCT01853423

Start Date

October 1 2013

End Date

July 1 2016

Last Update

August 19 2016

Active Locations (1)

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1

LeBonheur Children's Hospital

Memphis, Tennessee, United States, 38103