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

This study has been completed.
Sponsor:
Collaborator:
Novartis
Information provided by:
Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT00208026
First received: September 13, 2005
Last updated: May 9, 2008
Last verified: May 2008

September 13, 2005
May 9, 2008
September 2005
March 2008   (final data collection date for primary outcome measure)
  • Blood concentration of pimecrolimus [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Eczema Area and Severity Index (EASI) [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Netherton Area and Severity Assessment (NASA) [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Investigator's Global Evaluation of Disease (IGED) [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Pruritus Severity Assessment [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Transepidermal water loss [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • 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
Complete list of historical versions of study NCT00208026 on ClinicalTrials.gov Archive Site
  • Complete blood count with differential [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Blood electrolytes and fasting glucose [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Blood urea nitrogen and creatinine [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Liver function tests [ Time Frame: Each visit ] [ Designated as safety issue: No ]
  • Complete blood count with differential
  • Blood electrolytes and fasting glucose
  • Blood urea nitrogen and creatinine
  • Liver function tests
Not Provided
Not Provided
 
Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome
Exploratory Safety and Systemic Absorption of Elidel (Pimecrolimus) 1% Cream for the Treatment of Netherton Syndrome

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.

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.

Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Netherton Syndrome
Drug: Elidel (Pimecrolimus) 1% Cream
Open label single arm
Other Names:
  • Elidel
  • Pimecrolimus 1% Cream
  • Eczema
1
Treatment with drug/Elidel.Single arm-open-label treatment arm. A Pilot Study of the Efficacy and Safety of Pimecrolimus Cream 1% for the Treatment of Netherton Syndrome:
Intervention: Drug: Elidel (Pimecrolimus) 1% Cream
Yan AC, Honig PJ, Ming ME, Weber J, Shah KN. The safety and efficacy of pimecrolimus, 1%, cream for the treatment of Netherton syndrome: results from an exploratory study. Arch Dermatol. 2010 Jan;146(1):57-62. doi: 10.1001/archdermatol.2009.326.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
3
March 2008
March 2008   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Clinical diagnosis of Netherton syndrome
  • Normal laboratory values within 3 months prior to enrollment
  • Signed written informed consent
  • Willingness and ability to comply with the study requirements
  • For women of childbearing age, negative urine pregnancy test at enrollment and then monthly thereafter; women of childbearing age who are not abstinent must use contraception.

Exclusion Criteria:

  • Clinically significant physical examination or laboratory abnormalities
  • Clinical evidence of liver disease or liver injury as documented by abnormal liver function tests
  • Symptoms of a significant acute illness in the 30 week period preceding the start of treatment
  • Patients with known serious adverse reactions or hypersensitivity to macrolides or calcineurin inhibitors or with known hypersensitivity to any of the ingredients of the study medication or history of adverse reactions to the anesthetic product used for blood draws
  • Topical tacrolimus or Elidel within 2 weeks prior to dosing
  • Systemic steroid, systemic tacrolimus, or any immunosuppressant within 1 month prior to dosing
  • Phototherapy within 1 month prior to dosing
  • Use of inhibitors of CYP3A4 iso-enzyme within 2 weeks prior to dosing
  • Topical steroids or other topical therapy (except tacrolimus) may be used up to the day of 1st application of Elidel; however, treatment must be discontinued during the treatment period. Topical treatment of corticosteroids may resume immediately after the treatment period or in case an alert value has been exceeded and the Elidel treatment will be continued only on the face and neck.
  • Participation in any clinical trials within 2 months prior to dosing
  • History or clinical evidence of cardiovascular, respiratory, renal, hepatic, gastrointestinal, hematologic, neurologic disease, or any disease other than Netherton syndrome, that may put the subject at undue risk. Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs.
  • History of presence of malignancy or lymphoproliferative disease
  • Presence of any viral or fungal or untreated bacterial skin infection
  • Known HIV positivity or active hepatitis B or C
  • History of immunocompromise
  • No vaccines containing live viruses are to be administered during the study period.
Both
2 Years to 18 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00208026
2004-11-4063
Yes
Albert C. Yan, M.D./Section Chief, Pediatric Dermatology, The Children's Hospital of Philadelphia
Children's Hospital of Philadelphia
Novartis
Principal Investigator: Albert C Yan, MD Children's Hospital of Philadelphia
Children's Hospital of Philadelphia
May 2008

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP