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Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

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ClinicalTrials.gov Identifier: NCT02152007
Recruitment Status : Completed
First Posted : June 2, 2014
Results First Posted : October 7, 2016
Last Update Posted : October 7, 2016
Sponsor:
Collaborator:
Pachyonychia Congenita Project
Information provided by (Responsible Party):
TransDerm, Inc.

Study Description
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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

Condition or disease Intervention/treatment Phase
Pachyonychia Congenita Drug: 1% sirolimus cream (TD201 1%) Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita
Study Start Date : January 2014
Actual Primary Completion Date : July 2015
Actual Study Completion Date : November 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Sirolimus

Arms and Interventions
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Arm Intervention/treatment
Experimental: Split-body 1% sirolimus cream (TD201 1%)
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.
 Drug: 1% sirolimus cream (TD201 1%)
1% sirolimus cream (TD201 1%)


Outcome Measures
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Primary Outcome Measures :
  1. Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels [ Time Frame: Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug ]
    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 :
  1. Weekly Assessments Recorded in the PC Quality of Life Index [ Time Frame: Weekly for 39 weeks ]
    Patient-reported weekly assessment in the PC Quality of Life Index

  2. Daily Assessments Recording in the PC Measurement Diary [ Time Frame: Weekly for 39 weeks ]

Other Outcome Measures:
  1. Investigator Assessment of Local Tolerability [ Time Frame: Prior to application of study drug and within 15-45 minutes after application of study drug at each visit for 39 weeks ]
    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

  2. Standardized Photographs [ Time Frame: Each study visit over 39 weeks ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02152007


Locations
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United States, California
Stanford University
Stanford, California, United States, 94305
Sponsors and Collaborators
TransDerm, Inc.
Pachyonychia Congenita Project
Investigators
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Study Chair: Roger L Kaspar, PhD TransDerm, Inc.
More Information
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Responsible Party: TransDerm, Inc.
ClinicalTrials.gov Identifier: NCT02152007    
Obsolete Identifiers: NCT02057614
Other Study ID Numbers: TD201 - CS01
FD-R-05116 ( Other Grant/Funding Number: Orphan Products Development (OPD) )
First Posted: June 2, 2014    Key Record Dates
Results First Posted: October 7, 2016
Last Update Posted: October 7, 2016
Last Verified: October 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by TransDerm, Inc.:
pachyonychia congenita
Additional relevant MeSH terms:
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Pachyonychia Congenita
Nails, Malformed
Pathological Conditions, Anatomical
Ectodermal Dysplasia
Abnormalities, Multiple
Congenital Abnormalities
Skin Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Nail Diseases
 Skin Diseases
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs