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
Detailed Summary:
Sponsor: TransDerm, Inc.
Current Primary Outcome: Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels [ Time Frame: Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug ]
Original Primary Outcome: Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels [ Time Frame: The change in sirolimus levels 2 weeks and then monthly over time for the average study duration of 25 weeks or within 2 weeks of the last dose of study drug ]
Current Secondary Outcome:
- Weekly Assessments Recorded in the PC Quality of Life Index [ Time Frame: Weekly for 39 weeks ]Patient-reported weekly assessment in the PC Quality of Life Index
- Daily Assessments Recording in the PC Measurement Diary [ Time Frame: Weekly for 39 weeks ]
Original Secondary Outcome:
- Weekly Assessments Recorded in the PC Quality of Life Index [ Time Frame: Participants will be followed for the duration of the study, an expected average of 39 weeks ]
- Daily Assessments Recording in the PC Measurement Diary [ Time Frame: Participants will be followed for the duration of the study, an expected average of 39 weeks ]
Information By: TransDerm, Inc.
Dates:
Date Received: February 5, 2014
Date Started: January 2014
Date Completion:
Last Updated: October 5, 2016
Last Verified: October 2016