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Trial record 2 of 2 for:    "Bullous Pemphigoid" | "Antineoplastic Agents, Hormonal"

Efficacy and Safety of Omalizumab in Bullous Pemphigoid

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ClinicalTrials.gov Identifier: NCT00472030
Recruitment Status : Completed
First Posted : May 10, 2007
Results First Posted : October 16, 2012
Last Update Posted : October 16, 2012
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Janet Fairley, University of Iowa

Tracking Information
First Submitted Date  ICMJE May 8, 2007
First Posted Date  ICMJE May 10, 2007
Results First Submitted Date  ICMJE June 26, 2012
Results First Posted Date  ICMJE October 16, 2012
Last Update Posted Date October 16, 2012
Study Start Date  ICMJE August 2007
Actual Primary Completion Date December 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 17, 2012)
  • 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 Measures  ICMJE
 (submitted: May 8, 2007)
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 ]
Change History Complete list of historical versions of study NCT00472030 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 17, 2012)
  • 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 Measures  ICMJE
 (submitted: May 8, 2007)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Omalizumab in Bullous Pemphigoid
Official Title  ICMJE 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 Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Bullous Pemphigoid
Intervention  ICMJE
  • Drug: Omalizumab
    Patients will be treated with 150-375 milligrams of Omalizumab (Xolair), based on their baseline weight and serum Immunoglobulin E levels. Omalizumab will be administered subcutaneously on Day 1, and on Week 2, 4, 6, 8, 10, 12 and 14 treatment.
    Other Name: Xolair
  • Drug: prednisone
    Prednisone, to a maximum dose of 0.5 mg/kg/day.
Study Arms  ICMJE
  • Experimental: Omalizumab
    Patients will be treated with 150-375 milligrams of Omalizumab (Xolair), based on their baseline weight and serum Immunoglobulin E levels. Omalizumab will be administered subcutaneously on Day 1, and on Week 2, 4, 6, 8, 10, 12 and 14 treatment.
    Intervention: Drug: Omalizumab
  • Active Comparator: Prednisone
    The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
    Intervention: Drug: prednisone
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 11, 2012)
2
Original Estimated Enrollment  ICMJE
 (submitted: May 8, 2007)
12
Actual Study Completion Date  ICMJE December 2010
Actual Primary Completion Date December 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Patients must have the clinical and histological findings consistent with bullous pemphigoid. Clinically this is defined as urticarial plaques and/or vesicles and bullae. Histologically patients must show characteristic eosinophilic spongiosis and/or subepidermal separation of the skin consistent with BP.
  • Patients must have either a positive direct (IgG and/or C3 at the BMZ) or indirect (IgG on the roof of salt-split skin) immunofluorescence microscopy features of pemphigoid.
  • Patients of both sexes, all races and ethnic backgrounds that are 18 years of age or older will be eligible to participate in this study.
  • Patients much have more than 5% total body surface involved, since patients with less extensive disease are often treated with topical measures only.

Exclusion Criteria:

  • Women of childbearing potential not using the contraception method(s) specified during this study. Women of childbearing potential must use proven birth control methods (such as - abstinence, birth control pills, intrauterine device, barrier method combined with gel or foam with spermicide, tubal ligation, or a partner who has had a vasectomy).
  • Women who are pregnant or breastfeeding.
  • Patients under the age of 18.
  • Patients unable to give informed consent.
  • Known sensitivity to study drug(s) or class of study drug(s).
  • Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study.
  • Any cancer other than non-melanoma skin cancer in the past 5 years.
  • All non-melanoma skin cancers must have been adequately treated at entrance to the study.
  • Use of any other investigational agent in the last 30 days.
  • Treatment with prednisone in the past 2 weeks.
  • Weight or serum IgE levels that place the patient outside standard dosing guidelines for Xolair.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00472030
Other Study ID Numbers  ICMJE 100569
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Janet Fairley, University of Iowa
Study Sponsor  ICMJE University of Iowa
Collaborators  ICMJE Genentech, Inc.
Investigators  ICMJE
Principal Investigator: Janet A Fairley, MD University of Iowa
PRS Account University of Iowa
Verification Date September 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP