Clinical trial NCT02152007: Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Official title: Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Pachyonychia Congenita
Keywords: pachyonychia congenita
Lead sponsor: TransDerm, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Pachyonychia Congenita Project
First posted: 2014-06-02
Enrollment: 15
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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

Primary outcomes:
measure: 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
Secondary outcomes:
measure: Weekly Assessments Recorded in the PC Quality of Life Index
timeFrame: Weekly for 39 weeks
measure: Daily Assessments Recording in the PC Measurement Diary
timeFrame: Weekly for 39 weeks
Other outcomes:
measure: Investigator Assessment of Local Tolerability
timeFrame: Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks
measure: Standardized Photographs
timeFrame: Each study visit over 39 weeks
Locations:
city: Stanford
country: United States
facility: Stanford University
state: California
Study officials:
affiliation: TransDerm, Inc.
name: Roger L Kaspar, PhD
role: STUDY_CHAIR
References: