Safety and Efficacy of Pimecrolimus Cream 1% in Atopic Disease Modification

An Investigation of the Safety and Efficacy of Elidel® 1% Cream in Atopic Disease Modification, Assessed in a 3-year Randomized Double-blind Vehicle Controlled Phase to Evaluate Effects on Atopic Dermatitis in Infants, and a 2-3 Year Open-label Phase to Evaluate the Effect of Early Intervention Versus Delayed Intervention With Elidel® on the Incidence of Asthma in Children

This study consists of a 3-year double-blind phase during which patients will receive atopic dermatitis (AD) treatment either with pimecrolimus cream 1% long-term management (LTM) or with a conventional corticosteroid-based treatment (1:1 ratio), followed by a 2 to 3-year open-label (OL) phase (all patients receiving pimecrolimus cream 1% LTM). At the end of the double-blind phase, the two treatment groups will be compared with respect to their efficacy in controlling AD; at the end of the OL phase, the incidence of asthma at the age of 6 years will be compared.

Study Overview

• Status: Terminated
• Conditions: Atopic Dermatitis
• Intervention / Treatment: Drug: Pimecrolimus; Drug: Corticosteroid

• Study Type: Interventional
• Enrollment (Actual): 1091
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35209
      • Alabama Allergy and Asthma Center
  • Arkansas
    • Fayetteville, Arkansas, United States, 72703
      • Northwest Arkansas Pediatric Clinic, P.A.
    • Jonesboro, Arkansas, United States, 72401
      • The Children's Clinic of Jonesboro
  • California
    • Mission Viejo, California, United States, 92691
      • Southern California Research
    • Orange, California, United States, 92868
      • Children's Hospital of Orange County
    • Redwood City, California, United States, 94063
      • Stanford Dermatology Clinic and Clinical Trials Dept
    • Sacramento, California, United States, 95819
      • Capital Allergy Respiratory Disease Center
    • San Diego, California, United States, 92123
      • Children's Hospital San Diego
    • Walnut Creek, California, United States, 94598
      • Allergy and Asthma Medical Group of Diablo Valley, Inc./ Clinical Research Division
  • Colorado
    • Denver, Colorado, United States, 80206
      • National Jewish Medical and Research Center
  • Florida
    • Coral Gables, Florida, United States, 33134
      • Dermatology Associates and Research
    • Pensacola, Florida, United States, 32503
      • Emerald Coast Clinical Research
  • Illinois
    • Chicago, Illinois, United States, 60614
      • Children's Memorial Hospital
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University Outpatient Clinical Research
  • Kentucky
    • Lexington, Kentucky, United States, 40509
      • Central Kentucky Research Associates
    • Louisville, Kentucky, United States, 40202
      • Dermatology Specialists
  • Louisiana
    • Metarie, Louisiana, United States, 70002
      • Rx R & D
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Children's Hospital - Boston
  • Michigan
    • Ann Arbor, Michigan, United States, 48103
      • Dermatology, PLLC
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic Rochester
  • Missouri
    • St Louis, Missouri, United States, 63117
      • Central Dermatology
  • Nebraska
    • Omaha, Nebraska, United States, 68144
      • Skin Specialists, P.C.
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Dartmouth-Hitchcock Medical Center/Dermatology Section
  • New York
    • Hopewell Junction, New York, United States, 12533
      • Children's Medical Group
    • New York, New York, United States, 10025
      • Dermatology Associates of St. Luke's - Roosevelt
  • Oregon
    • Gresham, Oregon, United States, 97030
      • Calcagno Research and Development
    • Portland, Oregon, United States, 97201
      • Oregon Health Science University - Dermatology Dept.
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • The Childrens' Hospital of Philadelphia
    • Pittsburgh, Pennsylvania, United States, 15212
      • Allegheny General Hospital
  • Tennessee
    • Nashville, Tennessee, United States, 37215
      • Tennessee Clinical Research Center
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital, BCM
  • Utah
    • Salt Lake City, Utah, United States, 84102
      • Alpine Medical Group, LLC
    • West Valley City, Utah, United States, 84120
      • Granger Medical Clinic
  • Virginia
    • Norfolk, Virginia, United States, 23507
      • Virginia Clinical Research
  • Washington
    • Seattle, Washington, United States, 98105
      • Asthma Inc

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 months to 1 year (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of atopic dermatitis
• Family history of atopy
• 3 to 18 months of age at baseline
• At least mild atopic dermatitis at baseline (investigator global assessment [IGA] greater or equal to 2)
• Clinical evidence of atopic dermatitis for no longer than 3 months

Exclusion Criteria:

• Diagnosis of or substantial clinical evidence for food or other allergies at baseline

Other protocol related criteria may apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 1; Pimecrolimus	Drug: Pimecrolimus; Pimecrolimus cream 1 %; Other Names: Elidel
Active Comparator: 2; Corticosteroid	Drug: Corticosteroid; conventional corticosteroid-based treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Atopic Dermatitis (AD) Disease Control Over 36 Months	Proportion of disease-free days in Step 2 or less (per Patient) using total number of days in study as the denominator- double-blind phase. Intent to Treat Population: defined as all randomized patients who were dispensed study medication and had at least one post baseline efficacy measurement.	36 months
Effect of Early Use of Pimecrolimus Cream 1% in Reducing the Incidence of Asthma at 6 Years of Age	Note: The results for this efficacy variable are not reported due to early termination of the study.	6 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long Term Safety in Infants and Young Children	Note: The results of this secondary outcome is not reported due to early termination of the study.	6 years
Incidence of Allergic Rhinitis, Allergic Conjunctivitis and Food Allergies	Percentage of Patients who had allergic rhinitis, allergic conjunctivitis and food allergies at the end of the 36 month double blind study. Note: The results at six years are not reported due to early termination of the study.	6 years (36 month Double-Blind Phase)
Corticosteroid and Pimecrolimus Drug Use	Corticosteroid and pimecrolimus study medication days of exposure during the 36 month double-blind phase. Note: Although the double-blind phase was designed to be 36 months (3 years) in length, the last double-blind visit for some patients occurred after 36 months.	48 months
Atopic Dermatitis (AD) Remission Time	Longest duration of atopic dermatitis (AD) remission during the 36 month double-blind treatment phase. A remission day was defined as a diary day with a positive response ("yes") to the question "No or almost no eczema?" and a response of no treatment except emollients to the question "Medication used".	36 month Double-Blind Phase
Patient/Caregiver Quality of Life	Change from Baseline in the total Parents' Index of Quality of Life-Atopic Dermatitis (PIQoL-AD) score in the double-blind phase. PIQoL-AD Score = (sum of valid items/number of valid items) * 28. Scores range from a minimum value of 0 to a maximum value of 28 with a high total overall score indicating poor quality of life.	From Baseline to Visit 5 , 6, 8, 10, 12, and 14

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Publications and helpful links

General Publications

• Spergel JM, Boguniewicz M, Schneider L, Hanifin JM, Paller AS, Eichenfield LF. Food Allergy in Infants With Atopic Dermatitis: Limitations of Food-Specific IgE Measurements. Pediatrics. 2015 Dec;136(6):e1530-8. doi: 10.1542/peds.2015-1444.

Study record dates

Study Major Dates

• Study Start: October 1, 2003
• Primary Completion (Actual): January 1, 2008
• Study Completion (Actual): November 1, 2008

Study Registration Dates

• First Submitted: July 26, 2005
• First Submitted That Met QC Criteria: July 26, 2005
• First Posted (Estimate): July 28, 2005

Study Record Updates

• Last Update Posted (Estimate): March 23, 2011
• Last Update Submitted That Met QC Criteria: February 25, 2011
• Last Verified: February 1, 2011

More Information

Terms related to this study

• Keywords: Atopic, dermatitis, asthma, children, modification; Atopic dermatitis/atopy
• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Genetic; Hypersensitivity; Skin Diseases, Eczematous; Dermatitis; Eczema; Dermatitis, Atopic; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Enzyme Inhibitors; Analgesics; Sensory System Agents; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Anti-Inflammatory Agents; Antirheumatic Agents; Immunosuppressive Agents; Immunologic Factors; Dermatologic Agents; Calcineurin Inhibitors; Pimecrolimus
• Other Study ID Numbers: CASM981CUS09