Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Trial ID or NCT#
Status
NOT 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
- 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
- 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
Investigator(s)
Contact us to find out if this trial is right for you.
Contact
Joyce Teng, MD, PhD
650-723-6493
View on ClinicalTrials.gov
