Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

Trial ID or NCT#

NCT02152007

Status

not recruiting iconNOT RECRUITING

Purpose

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

Official Title

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

Eligibility Criteria

Ages Eligible for Study: Older than 16 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. Subjects must:
    1. * 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:
  1. A Subject with any of the following criteria is not eligible for inclusion in this study:
    1. * 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)
  2. Prior and Current Treatment
    1. * 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
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Investigator(s)

Joyce Teng, MD, PhD
Joyce Teng, MD, PhD
Dermatologist, Pediatric dermatologist
Professor of Dermatology and, by courtesy, of Pediatrics

Contact us to find out if this trial is right for you.

Contact

Joyce Teng, MD, PhD
650-723-6493

View on ClinicalTrials.gov