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Efficacy and Safety of Omalizumab in Bullous Pemphigoid

This study has been completed.
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Janet Fairley, University of Iowa
ClinicalTrials.gov Identifier:
NCT00472030
First received: May 8, 2007
Last updated: September 17, 2012
Last verified: September 2012
  Purpose

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.


Condition Intervention Phase
Bullous Pemphigoid
Drug: Omalizumab
Drug: prednisone
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Case Series on the Effects of Xolair (Omalizumab) in Bullous Pemphigoid

Resource links provided by NLM:


Further study details as provided by University of Iowa:

Primary Outcome Measures:
  • Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters. [ Time Frame: Up to 24 weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Decrease in Anti-BP180 IgG (Immunoglobulin G Anti-Bullous Pemphigoid 180 Antibody) Following Treatment With Omalizumab. [ Time Frame: Up to 24 weeks ] [ Designated as safety issue: No ]
    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. ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
  • Change in Histamine Release Assay Following Treatment With Omalizumab. [ Time Frame: Up to 24 weeks ] [ Designated as safety issue: No ]
    The histamine release assay measures the release of histamine which occurs upon stimulation of basophilic granulocytes depending upon their sensitivity to an allergen.


Enrollment: 2
Study Start Date: August 2007
Study Completion Date: December 2010
Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
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
Active Comparator: Prednisone
The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.
Drug: prednisone
Prednisone, to a maximum dose of 0.5 mg/kg/day.
Other Name: Prednisone

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00472030

Locations
United States, Iowa
University of Iowa, Department of Dermatology
Iowa City, Iowa, United States, 52242
Sponsors and Collaborators
University of Iowa
Genentech, Inc.
Investigators
Principal Investigator: Janet A Fairley, MD University of Iowa
  More Information

Publications:
Responsible Party: Janet Fairley, Professor, University of Iowa
ClinicalTrials.gov Identifier: NCT00472030     History of Changes
Other Study ID Numbers: 100569
Study First Received: May 8, 2007
Results First Received: June 26, 2012
Last Updated: September 17, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Iowa:
bullous pemphigoid
omalizumab
xolair
IgE
autoimmunity
skin disease

Additional relevant MeSH terms:
Pemphigoid, Bullous
Autoimmune Diseases
Immune System Diseases
Skin Diseases
Skin Diseases, Vesiculobullous
Omalizumab
Prednisone
Anti-Allergic Agents
Anti-Asthmatic Agents
Anti-Inflammatory Agents
Antineoplastic Agents
Antineoplastic Agents, Hormonal
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Pharmacologic Actions
Physiological Effects of Drugs
Respiratory System Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014