Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC) (NCT02152007) | Clinical Trial Compass
CompletedPhase 1
Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
United States15 participantsStarted 2014-01
Plain-language 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
Who can participate
Age range
16 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels
Timeframe: Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug