Efficacy and Safety of Omalizumab in Bullous Pemphigoid

NCT00472030 | Completed Phase 4 Results

• 1 other identifier: 100569
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Bullous Pemphigoid

Keywords

bullous pemphigoid; omalizumab; xolair; IgE; autoimmunity; skin disease

Outcome Measures

Primary Outcomes (3)

• Median Time From First Dose of Omalizumab Treatment to Cessation of New Blisters.

  The study subject underwent physical examination and was assessed for cessation of new blister formation via physical examination and photography.

  Up to 24 weeks

• Percent Decrease in the Total Body Surface Area Affected By Active Bullous Pemphigoid Skin Disease From Day 0 to Week 24.

  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).

  Up to 24 weeks

• Median Increase in Prednisone Dosage Measured at Week 4, 8 and 24 in Patients Treated With Omalizumab and in Patients Receiving Standard Therapy.

  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.

  Week 4, Week 8 and Week 24

Secondary Outcomes (4)

• Decrease in Anti-BP180 IgG (Immunoglobulin G Anti-Bullous Pemphigoid 180 Antibody) Following Treatment With Omalizumab.

  Up to 24 weeks

• Decrease in Eosinophil Levels Following Treatment With Omalizumab.

  Baseline, 24 weeks.

• Decrease in Anti-BP230 Antibody IgG (Anti-bullous Pemphigoid 230 Antibody Immunoglobulin G) At Baseline and Week 16

  Up to 24 weeks

• Change in Histamine Release Assay Following Treatment With Omalizumab.

  Up to 24 weeks

Study Arms (2)

Omalizumab

EXPERIMENTAL

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

Prednisone

ACTIVE COMPARATOR

The control arm of the study will receive standard prednisone therapy to a maximum dose of 0.5 mg/kg/day.

Drug: prednisone

Interventions

Omalizumab DRUG

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.

Also known as: Xolair

Omalizumab

prednisone DRUG

Prednisone, to a maximum dose of 0.5 mg/kg/day.

Prednisone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Iowa: lead
• Genentech, Inc.: collaborator

Study Sites (1)

University of Iowa, Department of Dermatology

Iowa City, Iowa, 52242, United States

Location

Related Publications (3)

• Dimson OG, Giudice GJ, Fu CL, Van den Bergh F, Warren SJ, Janson MM, Fairley JA. Identification of a potential effector function for IgE autoantibodies in the organ-specific autoimmune disease bullous pemphigoid. J Invest Dermatol. 2003 May;120(5):784-8. doi: 10.1046/j.1523-1747.2003.12146.x.

  PMID: 12713582 BACKGROUND

• Fairley JA, Fu CL, Giudice GJ. Mapping the binding sites of anti-BP180 immunoglobulin E autoantibodies in bullous pemphigoid. J Invest Dermatol. 2005 Sep;125(3):467-72. doi: 10.1111/j.0022-202X.2005.23853.x.

  PMID: 16117787 BACKGROUND

• Holgate ST, Djukanovic R, Casale T, Bousquet J. Anti-immunoglobulin E treatment with omalizumab in allergic diseases: an update on anti-inflammatory activity and clinical efficacy. Clin Exp Allergy. 2005 Apr;35(4):408-16. doi: 10.1111/j.1365-2222.2005.02191.x.

  PMID: 15836747 BACKGROUND

MeSH Terms

Conditions

Pemphigoid, Bullous; Autoimmune Diseases; Skin Diseases

Interventions

Omalizumab; Prednisone

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin and Connective Tissue Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Anti-Idiotypic; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Serum Globulins; Globulins; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Limitations and Caveats

Limitations of this trial include low enrollment of research subjects and the need to withdraw a subject from the trial.

Results Point of Contact

• Title: Janet Fairley, M.D.
• Organization: University of Iowa

janet-fairley@uiowa.edu

319-384-8560

Study Officials

• Janet A Fairley, MD

  University of Iowa

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 8, 2007
• First Posted: May 10, 2007
• Study Start: August 1, 2007
• Primary Completion: December 1, 2010
• Study Completion: December 1, 2010
• Last Updated: October 16, 2012
• Results First Posted: October 16, 2012

Record last verified: 2012-09

Locations

US (1)