Overview

Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

Status:
Completed
Trial end date:
2016-07-01
Target enrollment:
0
Participant gender:
All
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 angiofibromas that would benefit from treatment.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Le Bonheur Children's Hospital
Collaborators:
Pfizer
University of Tennessee Health Science Center
Treatments:
Everolimus
Sirolimus
Criteria
Inclusion Criteria:

- 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.

1. Patient must have a diagnosis of Tuberous Sclerosis Complex.

2. Female or male patients over the age of 3 years.

3. 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 Criteria:

- Patients will be excluded from entry into the study if any of the following are true:

1. Patient has a history of drug allergy to rapamune.

2. Patient is pregnant or lactating.

3. Subject is receiving therapy with Rapamycin.

4. Subject is receiving any form of immunosuppression or has previously experienced
immune dysfunction.

5. Subject is currently participating in or has participated within the Last 30 days
in a clinical trial involving an investigative drug.

6. Subject has other dermatologic conditions that would preclude or prevent adequate
assessment of changes to their facial angiofibromas.

7. Subject has had laser surgery, cryotherapy, or other dermatologic treatment to
their facial angiofibromas within the previous 6 months.