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 ]

The primary outcome measure for this Phase 1b safety study is evaluation of system absorption through measurement of serum sirolimus trough levels. The limit of detection of the assay was 2.0 ng/mL.


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