Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Completed · Phase 1
Conditions studied: Pachyonychia Congenita
In brief
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
Key facts
- Study ID
- NCT02152007
- Run by
- TransDerm, Inc.
- People needed
- 15
- Starts
- 2014-01-01
- Expected to finish
- 2015-11-01
- Last updated by the study team
- 2016-10-07
Who can join
Age: 16 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Stanford University — Stanford, California, United States
Full record on ClinicalTrials.gov
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