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Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Not Recruiting
Trial ID: NCT02152007
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
Stanford Investigator(s)
Joyce Teng, MD, PhD
Professor of Dermatology and, by courtesy, of Pediatrics
Eligibility
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
Intervention(s):
drug: 1% sirolimus cream (TD201 1%)
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Joyce Teng, MD, PhD
650-723-6493