Abstract | BACKGROUND AND OBJECTIVES: PATIENTS AND METHODS: The study was a prospective, randomized, double-blind, placebo-controlled study performed at the University of Texas Health Science Center at Houston. Study subjects were recruited from the patient populations at the University of Texas Tuberous Sclerosis Center of Excellence. All subjects were over the age of 13 years and had a diagnosis of tuberous sclerosis complex. Subjects were excluded if they were using any form of rapamycin or if they were pregnant. Study subjects applied the study product to their facial angiofibromas nightly for a duration of 6 months. The investigational product contained one of three doses of rapamycin compounded with Skincerity®: (i) no rapamycin; (ii) 1 mg of rapamycin per 30 cc (0.003%); or (iii) 5 mg of rapamycin per 30 cc (0.015%). Plasma rapamycin concentrations were measured monthly to test for systemic absorption. Complete blood counts were performed monthly to test for anemia, neutropenia, or thrombocytopenia. Upon completion of the trial, subjects were asked if the formulation had improved the appearance of their facial angiofibromas. RESULTS: Twenty-three subjects completed the study. There was no detectable systemic absorption of rapamycin (all blood concentrations were <1.0 ng/mL). There were no significant changes in white blood cell, red blood cell, or platelet counts. Seventy-three percent of subjects in the treatment arms versus 38% of subjects in the placebo arm reported a subjective improvement in the appearance of their facial angiofibromas. CONCLUSION: TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT01031901.
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Authors | Mary Kay Koenig, Adelaide A Hebert, Joan Roberson, Joshua Samuels, John Slopis, Audrey Woerner, Hope Northrup |
Journal | Drugs in R&D
(Drugs R D)
Vol. 12
Issue 3
Pg. 121-6
(Sep 01 2012)
ISSN: 1179-6901 [Electronic] New Zealand |
PMID | 22934754
(Publication Type: Clinical Trial, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
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Chemical References |
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Topics |
- Administration, Topical
- Adolescent
- Adult
- Angiofibroma
(drug therapy)
- Double-Blind Method
- Facial Neoplasms
(drug therapy)
- Female
- Humans
- Male
- Prospective Studies
- Sirolimus
(administration & dosage, adverse effects)
- Tuberous Sclerosis
(drug therapy, pathology)
- Young Adult
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