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Sponsors and Collaborators: |
Children's Hospital of Philadelphia Novartis |
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Information provided by: | Children's Hospital of Philadelphia |
ClinicalTrials.gov Identifier: | NCT00208026 |
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 | Intervention | Phase |
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Netherton Syndrome |
Drug: Elidel (Pimecrolimus) 1% Cream |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Exploratory Safety and Systemic Absorption of Elidel (Pimecrolimus) 1% Cream for the Treatment of Netherton Syndrome |
Enrollment: | 3 |
Study Start Date: | September 2005 |
Study Completion Date: | March 2008 |
Primary Completion Date: | March 2008 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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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:
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Drug: Elidel (Pimecrolimus) 1% Cream
Open label single arm
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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.
Ages Eligible for Study: | 2 Years to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 |
Principal Investigator: | Albert C Yan, MD | Children's Hospital of Philadelphia |
Responsible Party: | The Children's Hospital of Philadelphia ( Albert C. Yan, M.D./Section Chief, Pediatric Dermatology ) |
Study ID Numbers: | 2004-11-4063 |
Study First Received: | September 13, 2005 |
Last Updated: | May 9, 2008 |
ClinicalTrials.gov Identifier: | NCT00208026 |
Health Authority: | United States: Food and Drug Administration |
Pimecrolimus Netherton syndrome Ichthyosis linearis circumflexa |
Anti-Inflammatory Agents Disease Immunologic Factors Physiological Effects of Drugs Immunosuppressive Agents Pharmacologic Actions Pathologic Processes Analgesics, Non-Narcotic Sensory System Agents |
Syndrome Therapeutic Uses Anti-Inflammatory Agents, Non-Steroidal Analgesics Peripheral Nervous System Agents Antirheumatic Agents Central Nervous System Agents Dermatologic Agents |