Phase 1
N=15
Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Pachyonychia Congenita
Bottom Line
View on ClinicalTrials.gov: NCT02152007 ↗Enrolled (actual)
15
Serious AEs
0.0%
Results posted
Oct 2016
Primary outcome: Primary: Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels — 15; 15; 15; 14 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- 1% sirolimus cream (TD201 1%) (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- TransDerm, Inc.
- Primary completion
- Jul 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels |
15; 15; 15; 14; 14; 14 | — |
| SECONDARY Weekly Assessments Recorded in the PC Quality of Life Index |
— | — |
| SECONDARY Daily Assessments Recording in the PC Measurement Diary |
— | — |
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
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT02152007). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.