Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome - Full Text View

Sponsors and Collaborators:	Children's Hospital of Philadelphia Novartis
Information provided by:	Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:	NCT00208026

Purpose

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
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

Resource links provided by NLM:

MedlinePlus related topics: Eczema

Drug Information available for: Pimecrolimus

U.S. FDA Resources

Further study details as provided by Children's Hospital of Philadelphia:

Primary Outcome Measures:

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 ]

Secondary Outcome Measures:

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 ]

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
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:	Drug: Elidel (Pimecrolimus) 1% Cream Open label single arm

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.

Eligibility

Ages Eligible for Study:	2 Years to 18 Years
Genders Eligible for Study:	Both
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 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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00208026

Locations

United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104

Sponsors and Collaborators

Children's Hospital of Philadelphia

Novartis

Investigators

Principal Investigator:

Albert C Yan, MD

Children's Hospital of Philadelphia

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

No publications provided

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 History of Changes
Health Authority:	United States: Food and Drug Administration

Study placed in the following topic categories:

Anti-Inflammatory Agents
Immunologic Factors
Analgesics, Non-Narcotic
Anti-Inflammatory Agents, Non-Steroidal
Pimecrolimus
Peripheral Nervous System Agents

Analgesics
Antirheumatic Agents
Immunosuppressive Agents
Ichthyosis Linearis Circumflexa
Netherton Syndrome

Additional relevant MeSH terms:

Anti-Inflammatory Agents
Disease
Immunologic Factors
Physiological Effects of Drugs
Pimecrolimus
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

ClinicalTrials.gov processed this record on August 30, 2009