Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

Pachyonychia Congenita Clinical Trial

Official title:

Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02152007
• Other study ID #: TD201 - CS01
• Secondary ID: FD-R-05116
• Status: Completed
• Phase: Phase 1
• First received: February 5, 2014
• Last updated: October 5, 2016
• Start date: January 2014
• Est. completion date: November 2015

Study information

• Verified date: October 2016
• Source: TransDerm, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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

Other known NCT identifiers

• NCT02057614

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: November 2015
• Est. primary completion date: July 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years and older

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Pachyonychia Congenita

Intervention

Drug:
• 1% sirolimus cream (TD201 1%)
1% sirolimus cream (TD201 1%)

Locations

Country	Name	City	State
United States	Stanford University	Stanford	California

Sponsors (2)

Lead Sponsor	Collaborator
TransDerm, Inc.	Pachyonychia Congenita Project

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Investigator Assessment of Local Tolerability	Investigator assessment of local tolerability at the application sites on the plantar surfaces will be evaluated by the Investigator according to a 4-point scale (0, 1, 2, or 3; none to severe) with regard to: erythema, pruritis, stinging/burning, and crusting/erosion	Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks	No
Other	Standardized Photographs	An expert in the disease who is blinded to the study treatment will read the photographs of the callus area taken at each study visit. The reader will assess changes to the calluses based on criteria such as blisters, cracks, small/large size, and red or bloody spots on the callus. Change in calluses will be reported for both the right and left foot.	Each study visit over 39 weeks	No
Primary	Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels	The primary outcome measure for this Phase 1b safety study is evaluation of system absorption through measurement of serum sirolimus trough levels. The limit of detection of the assay was 2.0 ng/mL.	Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug	Yes
Secondary	Weekly Assessments Recorded in the PC Quality of Life Index	Patient-reported weekly assessment in the PC Quality of Life Index	Weekly for 39 weeks	No
Secondary	Daily Assessments Recording in the PC Measurement Diary		Weekly for 39 weeks	No