Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

• Status: Completed
• Trial end date: 2015-11-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: TransDerm, Inc.
• Collaborator: Pachyonychia Congenita Project
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

Subjects must:

• Be capable of understanding the purpose and risks of the study and sign a written Informed Consent Form (ICF)

• Be male or female ≥ 16 years of age at the time of the screening visit

• Have a confirmed diagnosis of PC by genotyping (e.g., familial) and clinically correlated painful keratoderma.

• Have roughly symmetrical calluses of similar severity on the plantar surface of both feet

• Women of childbearing potential must have a negative serum pregnancy test

• Subjects, whether male or female, with reproductive potential and who are sexually active must agree to use double-barrier contraception methods

Exclusion Criteria:

A Subject with any of the following criteria is not eligible for inclusion in this study:

• Use of other investigational drugs within 30 days of the screening visit and/or has not recovered from any side effects of prior investigational drugs or procedure in the affected area (e.g., a biopsy)

• Significant condition in the dermatologic treatment area such as trauma, or nonhealing chronic wound

• Pregnant or nursing (lactating) female, or a positive serum pregnancy test

• Active infection either systemic or local near the site of treatment requiring chronic or prolonged use of antimicrobial agents

• Known immunodeficiency including: Hepatitis A; Hepatitis B; Hepatitis C; Human Immunodeficiency Virus (HIV)

Prior and Current Treatment

• Unable to be discontinued from drugs known to either be inducers or inhibitors of CYP3A4/5 enzymes

• Unable to be discontinued from drugs known to be P-glycoprotein inhibitors