Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis
Status:
Completed
Trial end date:
2017-04-25
Target enrollment:
Participant gender:
Summary
The aim of the study is to determine the effect and safety of topical rapamycin or calcitriol
and their combination for the treatment of TSC-associated facial angiofibroma.
Methods: A total of 52 TSC patients including 20 male and 32 female subjects were recruited,
and 50 of them completed the period 1 study. In period 1, topical rapamycin (0.1%) or
calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day
by a left-right randomized, split-face comparison for 12 weeks. The primary end point was the
reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule
size, elevation and extension of the lesions at week 12. In period 2, the patients entered an
open-label study and were reassigned to use the more effective ointment on both cheeks for
another 12 weeks (week 13-24). A follow-up FASI analysis for recurrence after drug
discontinuance for 12 weeks was also performed (week 36). The secondary end point was the
reduction of Visual Analysis Score (VAS) evaluated by the subjects themselves at week 12.