Clinical Trial: Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional




Official Title: Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita

Brief Summary: A study to evaluate safety and efficacy of topical sirolimus to treat plantar keratoderma in adults with PC. Subjects may receive either placebo or treatment with at least 1 foot receiving topical sirolimus at some time. For certain phases of the study treatment assignment to the right and left foot will be randomized in a double blind fashion. Blood levels will test systemic absorption of sirolimus. Other safety and efficacy measures will be taken through the 39-week study duration. Funding Source - FDA OOPD