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

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ClinicalTrials.gov Identifier: NCT00208026
Recruitment Status : Completed
First Posted : September 21, 2005
Results First Posted : December 12, 2014
Last Update Posted : November 24, 2017
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Albert Yan, Children's Hospital of Philadelphia

Study Description
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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.

Condition or disease Intervention/treatment Phase
Netherton Syndrome Drug: Pimecrolimus 1% Cream Phase 1 Phase 2

Detailed Description:
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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Exploratory Safety and Systemic Absorption of Elidel (Pimecrolimus) 1% Cream for the Treatment of Netherton Syndrome
Study Start Date : September 2005
Actual Primary Completion Date : March 2008
Actual Study Completion Date : March 2008

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Pimecrolimus 1% Cream
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:
 Drug: Pimecrolimus 1% Cream
Open label single arm
Other Names:
  • Elidel
  • Eczema


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. Eczema Area and Severity Index (EASI) [ Time Frame: Each visit ]
  3. Netherton Area and Severity Assessment (NASA) [ Time Frame: Each visit ]
  4. Investigator's Global Evaluation of Disease (IGED) [ Time Frame: Each visit ]
  5. Pruritus Severity Assessment [ Time Frame: Each visit ]
  6. Transepidermal Water Loss [ Time Frame: Each visit ]

Secondary Outcome Measures :
  1. Complete Blood Count With Differential [ Time Frame: Each visit ]
  2. Blood Electrolytes and Fasting Glucose [ Time Frame: Each visit ]
  3. Blood Urea Nitrogen and Creatinine [ Time Frame: Each visit ]
  4. Liver Function Tests [ Time Frame: Each visit ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 Cytochrome P450 3A4 (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.
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00208026


Locations
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Children's Hospital of Philadelphia
Novartis Pharmaceuticals
Investigators
Principal Investigator: Albert C Yan, MD Children's Hospital of Philadelphia
More Information
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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:
Children's Hospital of Philadelphia  This link exits the ClinicalTrials.gov site
Foundation for Ichthyosis and Related Skin Types  This link exits the ClinicalTrials.gov site

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Albert Yan, Principal Investigator, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00208026     History of Changes
Other Study ID Numbers: 2004-11-4063
First Posted: September 21, 2005    Key Record Dates
Results First Posted: December 12, 2014
Last Update Posted: November 24, 2017
Last Verified: October 2017

Additional relevant MeSH terms:
Syndrome
Netherton Syndrome
Disease
Pathologic Processes
Abnormalities, Multiple
Congenital Abnormalities
Ichthyosiform Erythroderma, Congenital
Ichthyosis
Skin Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Keratosis
Skin Diseases
Pimecrolimus
 Tacrolimus
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Dermatologic Agents
Immunosuppressive Agents
Immunologic Factors
Calcineurin Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action