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

This study has been completed.
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Albert Yan, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT00208026
First received: September 13, 2005
Last updated: December 6, 2014
Last verified: December 2014
Results First Received: March 9, 2011  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Netherton Syndrome
Intervention: Drug: Pimecrolimus 1% Cream

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations

This was an open-label,single-arm study to investigate the safety profile of topical pimecrolimus cream.

Patients meeting the criteria for diagnosis of Netherton Syndrome were enrolled over a period of 3 years, from September 2005 through March 2008. The study was conducted in my medical office within The Children's Hospital of Philadelphia.


Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The eligibility was for male and female patients aged 2 to 18 years.They were required to have normal laboratory values within 3 months prior to enrollment. A 4 week washout period was required for systemic steroid, tacrolimus, immunosuppressives, phototherapy, and inhibitors of Cytochrome. 2 week washout for Isoenzyme,tacrolimus and pimecrolimus.

Reporting Groups
  Description
Elidel (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:

Participant Flow:   Overall Study
    Elidel (Pimecrolimus) 1% Cream  
STARTED     3  
COMPLETED     3  
NOT COMPLETED     0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Elidel (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:

Baseline Measures
    Elidel (Pimecrolimus) 1% Cream  
Number of Participants  
[units: participants]
  3  
Age  
[units: participants]
 
<=18 years     3  
Between 18 and 65 years     0  
>=65 years     0  
Gender  
[units: participants]
 
Female     3  
Male     0  
Region of Enrollment  
[units: participants]
 
United States     3  



  Outcome Measures

1.  Primary:   Highest Peak Blood Concentration of Pimecrolimus Over All Study Visits   [ Time Frame: Each visit up to 18 months ]

2.  Primary:   Eczema Area and Severity Index (EASI)   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

3.  Primary:   Netherton Area and Severity Assessment (NASA)   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

4.  Primary:   Investigator's Global Evaluation of Disease (IGED)   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

5.  Primary:   Pruritus Severity Assessment   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

6.  Primary:   Transepidermal Water Loss   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

7.  Secondary:   Complete Blood Count With Differential   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

8.  Secondary:   Blood Electrolytes and Fasting Glucose   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

9.  Secondary:   Blood Urea Nitrogen and Creatinine   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

10.  Secondary:   Liver Function Tests   [ Time Frame: Each visit ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
There were no early termination for any of the subjects.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Albert C. Yan, M.D.
Organization: The Children's Hospital of Philadelphia
phone: (215) 590-2158
e-mail: Yana@email.chop.edu


No publications provided by Children's Hospital of Philadelphia

Publications automatically indexed to this study:

Responsible Party: Albert Yan, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00208026     History of Changes
Other Study ID Numbers: 2004-11-4063
Study First Received: September 13, 2005
Results First Received: March 9, 2011
Last Updated: December 6, 2014
Health Authority: United States: Food and Drug Administration