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Simvastatin Treatment of Pachyonychia Congenita

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified October 2009 by Tel-Aviv Sourasky Medical Center.
Recruitment status was:  Not yet recruiting
Sponsor:
ClinicalTrials.gov Identifier:
NCT01382511
First Posted: June 27, 2011
Last Update Posted: June 27, 2011
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Tel-Aviv Sourasky Medical Center
  Purpose

Simvastatin and related statins, such as compactin, are able to inhibit the constitutive expression of inducible/repressible keratin genes such as K6a and K17. This effect is due to the reported ability of statins to inhibit STAT1 expression (Lee et al., 2007). The constitutive K6a promoter activity is dependent on STAT1 and blocked by simvastatin or siRNA against STAT1. This STAT1-dependent constitutive expression of K6a, is independent of JAK (Janus family of kinases).

Thus simvastatin is capable of down-regulating both the constitutive and interferon-inducible expression of PC-related keratins such as K6a and K17. Therefore, this class of molecule has potential for the treatment of PC or related inherited disorders where the causative mutation resides in an inducible/repressible keratin gene such as K6a or K17.

Simvastatin or other statins approved for human use might be delivered either orally, as is currently the case for cholesterol-lowering treatment or, if higher therapeutic doses are required in skin for reduction of hyperkeratosis in PC or in related keratinizing disorders, this might be achieved by topical formulations.


Condition Intervention
Pachyonychia Congenita Drug: Simvastatine

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Simvastatin Treatment of Pachyonychia Congenita

Resource links provided by NLM:


Further study details as provided by Tel-Aviv Sourasky Medical Center:

Primary Outcome Measures:
  • Assessment of decreased hyperkeratosis [ Time Frame: six months ]
    Decreased hyperkeratosis


Estimated Enrollment: 10
Study Start Date: July 2011
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Simvastatine
    80 mg
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with pachyonychia congenita with known mutations in keratin 6a. -

Exclusion Criteria:

  • The use of concomitant medications known to interact with simvastatin.
  • This would include itraconazole, ketoconazole, fluconazole, gemfibrozil, verapamil, diltiazem, mibefradil, erythromycin, clarithromycin, telithromycin, cyclosporine, ritonavir, nefazodone, danazol, amiodarone, Rifampin, and carbamazepine.
  • During the study subjects must agree to avoid grapefruit juice.
  • Also excluded are patients with a past history of myopathy, or impaired liver function.
  Contacts and Locations
No Contacts or Locations Provided
  More Information

Responsible Party: Department of Dermatology, Tel Aviv Sourasky Medical Center
ClinicalTrials.gov Identifier: NCT01382511     History of Changes
Other Study ID Numbers: 0462-09-TLV
First Submitted: June 14, 2011
First Posted: June 27, 2011
Last Update Posted: June 27, 2011
Last Verified: October 2009

Additional relevant MeSH terms:
Ectodermal Dysplasia
Nails, Malformed
Pachyonychia Congenita
Pathological Conditions, Anatomical
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Nail Diseases
Skin Diseases
Simvastatin
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors