Clinical Trial: Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Exploratory Safety and Systemic Absorption of Elidel (Pimecrolimus) 1% Cream for the Treatment of Netherton Syndrome

Brief Summary: Netherton syndrome is a genetic condition that can result in abnormal skin functioning. People with this condition often have red and scaling skin; sparse or short hair; and problems with absorption of medicines or chemicals that are applied to the skin. If these chemicals are absorbed at a high level, they may cause health problems. Elidel (pimecrolimus) is a new medicine that is available as a cream. It has been shown to help improve the appearance of the skin in patients with another skin condition known as atopic dermatitis, and is approved by the United States (US) Food and Drug Administration for use in children with mild to moderate atopic dermatitis. The purpose of this study is to determine if Elidel is safe, to see whether the medication is absorbed through the skin, and to see if side effects are associated with its use in children with Netherton syndrome.

Detailed Summary: Patients with Netherton syndrome, a rare genodermatosis, manifest a chronic, eczematous dermatitis with erythema and scaling that is often recalcitrant to conventional therapy with emollients and topical corticosteroids. These patients display an altered epidermal barrier with increased permeability to topical agents and are therefore susceptible to evaporative transepidermal water loss and infection. Topical therapy with the calcineurin inhibitors tacrolimus and pimecrolimus has been demonstrated to improve the skin integrity and the quality of life of patients with several chronic dermatoses, including atopic dermatitis. As a result of the underlying skin barrier dysfunction, however, the possibility of significant systemic absorption and resultant side effects is a concern when these agents are used in patients with Netherton syndrome. Experience with topical tacrolimus 0.1% ointment for patients with Netherton syndrome has demonstrated both marked efficacy as well as significant systemic absorption of the drug in this patient population. Use of topical pimecrolimus in patients with Netherton syndrome has not been reported to date. Investigation of the extent of systemic absorption and side effects will help to define the safety and efficacy profile of topical pimecrolimus in patients with Netherton syndrome.
Sponsor: Children's Hospital of Philadelphia

Current Primary Outcome:

  • Highest Peak Blood Concentration of Pimecrolimus Over All Study Visits [ Time Frame: Each visit up to 18 months ]
    At each scheduled visit, blood concentration of pimecrolimus were obtained. This value reflects the amount of pimecrolimus in the blood. This is measured directly from the blood and provides an estimate of the degree of absorption of the treatment medication through the skin into the blood. Analysis was performed as intent-to-treat with last value carried forward, but no data points were missing.
  • Eczema Area and Severity Index (EASI) [ Time Frame: Each visit ]
  • Netherton Area and Severity Assessment (NASA) [ Time Frame: Each visit ]
  • Investigator's Global Evaluation of Disease (IGED) [ Time Frame: Each visit ]
  • Pruritus Severity Assessment [ Time Frame: Each visit ]
  • Transepidermal Water Loss [ Time Frame: Each visit ]


Original Primary Outcome:

  • Blood concentration of pimecrolimus
  • Eczema Area and Severity Index (EASI)
  • Netherton Area and Severity Assessment (NASA)
  • Investigator's Global Evaluation of Disease (IGED)
  • Pruritus Severity Assessment
  • Transepidermal Water Loss


Current Secondary Outcome:

  • Complete Blood Count With Differential [ Time Frame: Each visit ]
  • Blood Electrolytes and Fasting Glucose [ Time Frame: Each visit ]
  • Blood Urea Nitrogen and Creatinine [ Time Frame: Each visit ]
  • Liver Function Tests [ Time Frame: Each visit ]


Original Secondary Outcome:

  • Complete Blood Count With Differential
  • Blood Electrolytes and Fasting Glucose
  • Blood Urea Nitrogen and Creatinine
  • Liver Function Tests


Information By: Children's Hospital of Philadelphia

Dates:
Date Received: September 13, 2005
Date Started: September 2005
Date Completion:
Last Updated: December 6, 2014
Last Verified: December 2014