Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

NCT01853423 | Completed Phase 1

• 2 other identifiers: UTHSC TSC0112-2008
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

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 angiofibromas that would benefit from treatment.

Conditions

Facial Angiofibroma

Keywords

Rapamune; Topical; Pediatric; Safety; Efficiency; Angiofibroma; Skin Cream

Outcome Measures

Primary Outcomes (1)

• Reduction of facial angiofibroma with use of rapamune facial skin cream

  1. Tolerability: Number of participants with adverse events 2. Efficacy: Skin assessment to measure improvement of facial angiofibromas 3. Safety: Rapamune levels (serum)

  1 year

Study Arms (1)

Rapamune

EXPERIMENTAL

Small amount of 0.1% rapamune ointment applied topically to affected facial areas twice daily for the first two weeks, then once daily.

Drug: Rapamune

Interventions

Rapamune DRUG

Small amount of 0.1% rapamune ointment applied topically to affected facial areas twice daily for the first two weeks, then once daily.

Also known as: Sirolimus

Rapamune

Eligibility Criteria

• Age: 3 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Le Bonheur Children's Hospital: lead
• Pfizer: collaborator
• University of Tennessee: collaborator

Study Sites (1)

LeBonheur Children's Hospital

Memphis, Tennessee, 38103, United States

Location

MeSH Terms

Conditions

Tuberous Sclerosis; Angiofibroma

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• James W Wheless, MD

  LeBonheur Children's Hospital- Neuroscience Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Doctor

Study Record Dates

• First Submitted: April 12, 2013
• First Posted: May 15, 2013
• Study Start: October 1, 2013
• Primary Completion: May 1, 2016
• Study Completion: July 1, 2016
• Last Updated: August 19, 2016

Record last verified: 2016-08

Locations

US (1)