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Status unknownNCT01382511Updated Jun 27, 2011

Simvastatin Treatment of Pachyonychia Congenita

An interventional study of Simvastatine in Pachyonychia Congenita, sponsored by Tel-Aviv Sourasky Medical Center. Status unknown. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2011-06-27.

Sponsored by Tel-Aviv Sourasky Medical Center · Not applicable, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Oct 2009), so the status shown — last known as Not yet recruiting — may be out of date.
Phase
Not applicable
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Pachyonychia Congenita
03

In context

Lead sponsor

Tel-Aviv Sourasky Medical Center is the lead sponsor of 541 studies on the registry; 49 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

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

Exclusion criteria

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.
05

Study design

Phase
Not applicable
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (estimated)

Interventions

  • DrugSimvastatine

    80 mg

06

What researchers measure

Primary outcomes

  1. Assessment of decreased hyperkeratosis

    Decreased hyperkeratosis

    Time frame: six months

07

Study locations

No study locations are listed for this record.

08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 27, 2011, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT01382511
Lead sponsor
Tel-Aviv Sourasky Medical Center
First posted
Jun 27, 2011
Start date
Jul 2011
Primary completion
Dec 2014 (estimated)
Last update
Jun 27, 2011
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Oct 2009. You cannot join it, but the record below documents what was studied.

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