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.