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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00208026
• Date of registration: 13/09/2005
• Prospective Registration: No
• Primary sponsor: Children's Hospital of Philadelphia
• Public title: Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome
• Scientific title: Exploratory Safety and Systemic Absorption of Elidel (Pimecrolimus) 1% Cream for the Treatment of Netherton Syndrome
• Date of first enrolment: September 2005
• Target sample size: 3
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00208026
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Albert C Yan, MD
• Affiliation: Children's Hospital of Philadelphia

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Netherton Syndrome

Intervention(s)

Drug: Pimecrolimus 1% Cream

Primary Outcome(s)

Blood Pimecrolimus Levels [Time Frame: Each visit up to 18 months: Study Days 1, 7, 14, 28, 56, 84, 175, 360, and 520]

Secondary ID(s)

2004-11-4063

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Novartis Pharmaceuticals

Results

• Results available: Yes
• Date Posted: 12/12/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00208026