Clinical Trial: Efficacy and Safety of Omalizumab in Bullous Pemphigoid

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open Label Case Series on the Effects of Xolair (Omalizumab) in Bullous Pemphigoid

Brief Summary:

The primary objective is to test the safety and efficacy of Xolair in the treatment of the autoimmune blistering disease, bullous pemphigoid (BP).

This is a pilot, open label case-control study. Patients treated with Xolair will be compared to patients receiving standard treatment with prednisone.

The enrollment period for the study is 24 weeks: 16 weeks active treatment and 8 additional weeks of observation.


Detailed Summary:

Objectives: The primary objective is to test the safety and efficacy of Omalizumab (Xolair) in the treatment of the autoimmune blistering disease, bullous pemphigoid (BP).

Study Rationale: The current treatment for bullous pemphigoid is non-specific immunosuppression, causing great morbidity in these patients. Recently, pathogenic Immunoglogulin Class E autoantibodies have been identified in these patients. Development of a more targeted approach to treatment may reduce morbidity.

Methodology: This is a pilot, open-label case-control study. Patients treated with Omalizumab (Xolair) will be compared to patients receiving standard treatment with prednisone.

Number of centers and patients: This is a single center study that will enroll 12 patients.

Population: Bullous pemphigoid patients, meeting clinical, histological and immunologic criteria for the disease will be enrolled. Pregnant women, children less than 18 years of age, and patients unable to give consent will be excluded from this preliminary study.

Investigational drug: Xolair® (Omalizumab)

Study duration: 24 weeks: 16 weeks of active treatment, 8 additional weeks of observation

Evaluation criteria: Primary: 1. Time to cessation of new blister formation. 2. Percent body surface area of skin involved before and after treatment 3. Total and average daily dose of prednisone required in 30, 60 and 180 days after starting Xolair. Secondary: 1. Number of circulating eosinophils 2. Measurement of circulating anti-BMZ (basement membrane zone) autoantibodies 3. Histamine release assay.


Sponsor: University of Iowa

Current Primary Outcome:

  • Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters. [ Time Frame: Up to 24 weeks ]
    The study subject underwent physical examination and was assessed for cessation of new blister formation via physical examination and photography.
  • Percent Decrease in the Total Body Surface Area Affected By Active Bullous Pemphigoid Skin Disease From Day 0 to Week 24. [ Time Frame: Up to 24 weeks ]
    Measurement of total body surface area affected by bullous pemphigoid active skin disease(active erosions, blisters, and/or lesions) was measured at Day 0 (prior to treatment with Omalizumab) and at 24 weeks (24 weeks is end of study).
  • Median Increase in Prednisone Dosage Measured at Week 4, 8 and 24 in Patients Treated With Omalizumab and in Patients Receiving Standard Therapy. [ Time Frame: Week 4, Week 8 and Week 24 ]
    The total dose of prednisone required to control the bullous pemphigoid at week 4, 8 and 24 weeks was to be calculated in both arms of this study.


Original Primary Outcome: Assessment of total and new blister numbers over the 16 week period. Time to the cessation of new blister formation. The total dose of prednisone (mg/kg/d) during the 16 week treatment period. Parameters will be compared to the controls. [ Time Frame: 24 total weeks ]

Current Secondary Outcome:

  • Decrease in Anti-BP180 IgG (Immunoglobulin G Anti-Bullous Pemphigoid 180 Antibody) Following Treatment With Omalizumab. [ Time Frame: Up to 24 weeks ]
    Anti-BP180 IgG levels were completed using an Elisa assay. Anti-BP180 IgG levels were obtained prior to baseline and at week 16
  • Decrease in Eosinophil Levels Following Treatment With Omalizumab. [ Time Frame: Baseline, 24 weeks. ]
    The subject's eosinophil count measured at baseline was compared to the eosinophil count at week 8. A normal eosinophil count at the University of Iowa Hospital lab is 0-0.4 cells per microliter
  • Decrease in Anti-BP230 Antibody IgG (Anti-bullous Pemphigoid 230 Antibody Immunoglobulin G) At Baseline and Week 16 [ Time Frame: Up to 24 weeks ]
  • Change in Histamine Release Assay Following Treatment With Omalizumab. [ Time Frame: Up to 24 weeks ]
    The histamine release assay measures the release of histamine which occurs upon stimulation of basophilic granulocytes depending upon their sensitivity to an allergen.


Original Secondary Outcome:

  • Circulating anti-BMZ autoantibodies by indirect immunofluorescence. Measure collagen XVII-specific IgE and IgG autoantibodies present in the sera of these patients at day 0, week 1,2,4,8,12,16, and 24. Specific antibodies will be measured by ELISA. [ Time Frame: 24 total weeks ]
  • An additional secondary assessment will be the number of circulating eosinophils and the ability of the patients' autoantibodies to trigger histamine release. These will be measured at the same time-points as the autoantibody measures. [ Time Frame: 24 total weeks ]


Information By: University of Iowa

Dates:
Date Received: May 8, 2007
Date Started: August 2007
Date Completion:
Last Updated: September 17, 2012
Last Verified: September 2012