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Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

Primary Purpose

Pachyonychia Congenita

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
1% sirolimus cream (TD201 1%)
Sponsored by
TransDerm, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pachyonychia Congenita focused on measuring pachyonychia congenita

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Stanford University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Split-body 1% sirolimus cream (TD201 1%)

Arm Description

This is a split-body design. Subjects will self-administer 1% topical sirolimus cream or placebo cream (no drug, vehicle control) on the plantar surface of each foot. At least one foot will be treated with topical sirolimus at some time during the study. Application will be one time daily for a total of 26 weeks. There will be an additional follow-up visit 3 months after the last application of study drug. The total duration of the study is 39 weeks.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Weekly Assessments Recorded in the PC Quality of Life Index
Patient-reported weekly assessment in the PC Quality of Life Index
Daily Assessments Recording in the PC Measurement Diary

Full Information

First Posted
February 5, 2014
Last Updated
October 5, 2016
Sponsor
TransDerm, Inc.
Collaborators
Pachyonychia Congenita Project
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1. Study Identification

Unique Protocol Identification Number
NCT02152007
Brief Title
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

2. Study Status

Record Verification Date
October 2016
Overall Recruitment Status
Completed
Study Start Date
January 2014 (undefined)
Primary Completion Date
July 2015 (Actual)
Study Completion Date
November 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
TransDerm, Inc.
Collaborators
Pachyonychia Congenita Project

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pachyonychia Congenita
Keywords
pachyonychia congenita

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Split-body 1% sirolimus cream (TD201 1%)
Arm Type
Experimental
Arm Description
This is a split-body design. Subjects will self-administer 1% topical sirolimus cream or placebo cream (no drug, vehicle control) on the plantar surface of each foot. At least one foot will be treated with topical sirolimus at some time during the study. Application will be one time daily for a total of 26 weeks. There will be an additional follow-up visit 3 months after the last application of study drug. The total duration of the study is 39 weeks.
Intervention Type
Drug
Intervention Name(s)
1% sirolimus cream (TD201 1%)
Intervention Description
1% sirolimus cream (TD201 1%)
Primary Outcome Measure Information:
Title
Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels
Description
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.
Time Frame
Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug
Secondary Outcome Measure Information:
Title
Weekly Assessments Recorded in the PC Quality of Life Index
Description
Patient-reported weekly assessment in the PC Quality of Life Index
Time Frame
Weekly for 39 weeks
Title
Daily Assessments Recording in the PC Measurement Diary
Time Frame
Weekly for 39 weeks
Other Pre-specified Outcome Measures:
Title
Investigator Assessment of Local Tolerability
Description
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
Time Frame
Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks
Title
Standardized Photographs
Description
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.
Time Frame
Each study visit over 39 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Roger L Kaspar, PhD
Organizational Affiliation
TransDerm, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

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