Efficacy and Safety of Omalizumab in Bullous Pemphigoid

University of Iowa

Status and phase: Completed; Phase 4

Conditions: Bullous Pemphigoid

Treatments: Drug: prednisone; Drug: Omalizumab

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00472030

100569 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 2 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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2 participants in 2 patient groups

Omalizumab

Experimental group

Description:

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.

Treatment:

Drug: Omalizumab

Prednisone

Active Comparator group

Description:

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

Treatment:

Drug: prednisone