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

Information source: Stanford University
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Pachyonychia Congenita; Plantar Keratoderma

Intervention: Topical Sirolimus (Drug)

Phase: Phase 1

Status: Recruiting

Sponsored by: Stanford University

Official(s) and/or principal investigator(s):
Joyce Teng, MD, PhD, Principal Investigator, Affiliation: Stanford University


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.

Clinical Details

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

Study design: Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Primary outcome:

Sirolimus Serum levels

Serum Sirolimus Levels


Minimum age: 16 Years. Maximum age: N/A. Gender(s): Both.


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

Locations and Contacts

Stanford University, Palo Alto, California 94305, United States; Recruiting
R Adeduntan, Phone: 650-721-7163
Joyce Teng, MD, PhD, Principal Investigator
Additional Information

Starting date: January 2014
Last updated: February 6, 2014

Page last updated: August 23, 2015

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