Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00208026 |
Recruitment Status :
Completed
First Posted : September 21, 2005
Results First Posted : December 12, 2014
Last Update Posted : August 14, 2019
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Condition or disease | Intervention/treatment | Phase |
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Netherton Syndrome | Drug: Pimecrolimus 1% Cream | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 3 participants |
Allocation: | N/A |
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 |

Arm | Intervention/treatment |
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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:
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Drug: Pimecrolimus 1% Cream
Open label single arm
Other Names:
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- Blood Pimecrolimus Levels [ Time Frame: Each visit up to 18 months: Study Days 1, 7, 14, 28, 56, 84, 175, 360, and 520 ]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.

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

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
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 |
Principal Investigator: | Albert C Yan, MD | Children's Hospital of Philadelphia |
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 |
Other Study ID Numbers: |
2004-11-4063 |
First Posted: | September 21, 2005 Key Record Dates |
Results First Posted: | December 12, 2014 |
Last Update Posted: | August 14, 2019 |
Last Verified: | July 2019 |
Netherton Syndrome 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 |
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 |