Topical Sirolimus for Plantar Keratoderma in Adults With Pachyonychia Congenita (PC)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Stanford University
TransDerm, Inc.
Pachyonychia Congenita Project
Information provided by (Responsible Party):
Stanford University Identifier:
First received: January 29, 2014
Last updated: February 6, 2014
Last verified: February 2014
The primary objective of this study is to assess the safety of topical sirolimus (TD201) 1% for plantar keratoderma for the treatment of pachyonychia congenita. This study would also like to assess the potential of sirolimus (TD201) to improve the clinical severity of plantar keratoderma, including pain.

Condition Intervention Phase
Pachyonychia Congenita
Plantar Keratoderma
Drug: Topical Sirolimus
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: A Prospective, Randomized, Double-Blind, Split-Body, Placebo-Controlled Evaluation of the Safety and Efficacy of Topical Sirolimus 1% (TD201) for Plantar Keratoderma in Adults With Pachyonychia Congenita

Resource links provided by NLM:

Further study details as provided by Stanford University:

Primary Outcome Measures:
  • Sirolimus Serum levels [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
    For the first 12 weeks of the study, sirolimus serum levels will be measured at monthly intervals (i.e., every 4 weeks) during the treatment phases of the study

  • Serum Sirolimus Levels [ Time Frame: 17-37 weeks ] [ Designated as safety issue: Yes ]
    For the last two visits (weeks 17-37), sirolimus serum levels will be measured at monthly intervals during the treatment phases of the study

Estimated Enrollment: 15
Study Start Date: January 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Topical Sirolimus Drug: Topical Sirolimus


Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subjects must

    1. Be capable of understanding the purpose and risks of the study and sign a written Informed Consent Form (ICF)
    2. Be male or female ≥ 16 years of age at the time of the screening visit
    3. Have a confirmed diagnosis of PC by genotyping (e.g., familial) and clinically correlated painful keratoderma.
    4. Have roughly symmetrical calluses of similar severity on the plantar surface of both feet
    5. Weigh at least 40 kg (88.2 lbs.)
    6. Have laboratory values within the range of normal
    7. Women of childbearing potential must have a negative serum pregnancy test on Day -14 and a negative urine pregnancy test within 48 hours prior to subject's assignment to study treatment at the baseline visit (Visit 1, Week 1, Day 1). All female subjects (including those with tubal ligations) will be considered to be of childbearing potential unless one or more of the following criteria are met:

      • Over age 60
      • Amenorrheic for at least 2 years if age 45-60 years
      • Have had a hysterectomy and/or bilateral oophorectomy
    8. Subjects with reproductive potential and who are sexually active must agree to use double-barrier contraception methods BEFORE beginning the study and for the duration of the study (37 weeks) or for 12 weeks after the last dose if study drug is discontinued prematurely

Exclusion Criteria:

  • A Subject with any of the following criteria is not eligible for inclusion in this study:

    1. Use of other investigational drugs within 30 days of the screening visit (for dermatologic drugs see below) and/or has not recovered from any side effects of prior investigational drugs or procedure in the affected area (e.g., a biopsy)
    2. Significant condition in the dermatologic treatment area such as trauma, which could impair evaluation for the treatment of PC or nonhealing chronic wound,
    3. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or history of serious allergic reaction; known allergy to sirolimus
    4. Prior history of grafting surgeries or other surgeries in the dermatologic treatment area
    5. Prior history of liver disease
    6. Poorly controlled hyperlipidemia
    7. Pregnant or nursing (lactating) female, or a positive serum pregnancy test on Day -14 and/or a positive urine pregnancy test within 48 hours of initiation of treatment
    8. Active infection (i.e. bacterial, viral, parasitic or fungal) either systemically or local near the site of treatment requiring chronic or prolonged use of antimicrobial agents
    9. Known immunodeficiency including:

      1. Hepatitis A
      2. Hepatitis B
      3. Hepatitis C
      4. Human Immunodeficiency Virus (HIV)
    10. Serious known concurrent medical illness or infection, or any medical condition that could potentially present a safety risk and/or prevent compliance with the requirements of the protocol
    11. Any other severe, acute or chronic medical or psychiatric condition (including current or history of substance abuse) or clinical laboratory abnormalities
    12. Current malignancy or a history of malignancy (within the previous 5 years)
    13. Myocardial infarction within 6 months prior to the baseline visit (Visit 1) or history of cardiac dysrhythmia E2. Prior and Current Treatment
    14. Unable to be discontinued from drugs known to either be inducers or inhibitors (moderate or strong) of cytochrome P450 isoenzymes
    15. Unable to be discontinued from drugs known to be strong or moderate P-glycoprotein (P-gp) inhibitors within 7 days prior to the Screening Visit
    16. Use of topical therapies on the dermatologic treatment area within 6 weeks prior to Screening visit (Day -14): pulsed dye laser, carbon dioxide laser, surgeries within the treatment area, topical sirolimus, topical retinoids, topical steroids or other topical prescription or over the counter medications
    17. Use of systemic drug therapies within 7 days prior to Screening Visit at Day -14: e.g., cytotoxic drugs, immunomodulators or immunosuppressants, or diltiazem, erythromycin, all azole antifungals, rifampin, nicardipine, verapamil
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02057614

United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94305
Contact: R Adeduntan    650-721-7163      
Principal Investigator: Joyce Teng, MD, PhD         
Sponsors and Collaborators
Stanford University
TransDerm, Inc.
Pachyonychia Congenita Project
Principal Investigator: Joyce Teng, MD, PhD Stanford University
  More Information

No publications provided

Responsible Party: Stanford University Identifier: NCT02057614     History of Changes
Other Study ID Numbers: TD201 - CS01
Study First Received: January 29, 2014
Last Updated: February 6, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Nail Diseases
Pachyonychia Congenita
Congenital Abnormalities
Ectodermal Dysplasia
Genetic Diseases, Inborn
Skin Abnormalities
Skin Diseases
Skin Diseases, Genetic
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on November 30, 2015