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NCT Number: NCT02152007

Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

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

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Key information

Conditions

Pachyonychia Congenita Abnormalities, Multiple Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Ectodermal Dysplasia Genetic Diseases, Inborn Nail Diseases Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

16 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Stanford University

Stanford, California, 94305, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

1% sirolimus cream (TD201 1%)

Drug

1% sirolimus cream (TD201 1%)

Primary outcomes

  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 outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

TransDerm, Inc.

Industry

Collaborators

  • Pachyonychia Congenita Project

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2015
Study completion
2015
First posted
Jun 2, 2014
Registry last updated
Oct 7, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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