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Simvastatin Treatment of Pachyonychia Congenita

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. Identifier: NCT01382511
Recruitment Status : Unknown
Verified October 2009 by Tel-Aviv Sourasky Medical Center.
Recruitment status was:  Not yet recruiting
First Posted : June 27, 2011
Last Update Posted : June 27, 2011
Information provided by:
Tel-Aviv Sourasky Medical Center

Brief Summary:

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 or disease Intervention/treatment Phase
Pachyonychia Congenita Drug: Simvastatine Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Simvastatin Treatment of Pachyonychia Congenita
Study Start Date : July 2011
Estimated Primary Completion Date : December 2014

Intervention Details:
  • Drug: Simvastatine
    80 mg

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

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

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.

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Responsible Party: Department of Dermatology, Tel Aviv Sourasky Medical Center Identifier: NCT01382511     History of Changes
Other Study ID Numbers: 0462-09-TLV
First Posted: June 27, 2011    Key Record Dates
Last Update Posted: June 27, 2011
Last Verified: October 2009
Additional relevant MeSH terms:
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Ectodermal Dysplasia
Pachyonychia Congenita
Nails, Malformed
Pathological Conditions, Anatomical
Abnormalities, Multiple
Congenital Abnormalities
Skin Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Nail Diseases
Skin Diseases
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors