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

NCT00208026 | Completed Phase 1 Results DMC

• 1 other identifier: 2004-11-4063
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Netherton Syndrome

Outcome Measures

Primary Outcomes (1)

• Blood Pimecrolimus Levels

  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.

  Each visit up to 18 months: Study Days 1, 7, 14, 28, 56, 84, 175, 360, and 520

Study Arms (1)

Pimecrolimus 1% Cream

EXPERIMENTAL

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

Interventions

Pimecrolimus 1% Cream DRUG

Open label single arm

Also known as: Elidel, Eczema

Pimecrolimus 1% Cream

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

• 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.

  PMID: 20083693 DERIVED

Related Links

• Children's Hospital of Philadelphia
• Foundation for Ichthyosis and Related Skin Types

MeSH Terms

Conditions

Netherton Syndrome

Interventions

pimecrolimus; WAS protein, human

Condition Hierarchy (Ancestors)

Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ichthyosiform Erythroderma, Congenital; Ichthyosis; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases

Limitations and Caveats

There were no early termination for any of the subjects.

Results Point of Contact

• Title: Albert C. Yan, M.D.
• Organization: The Children's Hospital of Philadelphia

Yana@email.chop.edu

(215) 590-2158

Study Officials

• Albert C Yan, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 13, 2005
• First Posted: September 21, 2005
• Study Start: September 1, 2005
• Primary Completion: March 1, 2008
• Study Completion: March 1, 2008
• Last Updated: August 14, 2019
• Results First Posted: December 12, 2014

Record last verified: 2019-07

Locations

US (1)