An Exploratory Study to Assess Multiple Doses of Omalizumab in Patients With Cystic Fibrosis Complicated by Acute Bronchopulmonary Aspergillosis (ABPA)

Study Overview
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Brief Summary
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This study will evaluate the safety and efficacy of omalizumab for the treatment of Allergic Bronchopulmonary Aspergillosis (ABPA) in patients with Cystic Fibrosis aged 12 years and older.

Official Title
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An Exploratory, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy of Multiple Doses of Omalizumab in Cystic Fibrosis Complicated by Allergic Bronchopulmonary Aspergillosis (ABPA)

Conditions
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Cystic Fibrosis, Allergic Bronchopulmonary Aspergillosis

Intervention / Treatment
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* Drug: Omalizumab
* Drug: Placebo
* Drug: Itraconazole


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Cystic Fibrosis complicated by Allergic Bronchopulmonary Aspergillosis (ABPA) Oral corticosteroid use for ABPA flare Age 12 years and older (except for Italy; ≥ 18 years) Total serum IgE levels ≥ 500 IU/mL Exclusion Criteria: History of cancer in the last 10 years. History of severe allergic reactions Pregnant and lactating women Prior use of Xolair Other protocol-defined inclusion/exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Omalizumab<br>Eligible participants received a maximum dose of 600 mg omalizumab via subcutaneous injection for 6 months in the double-blind phase of the study. The study medication was to be administered at the same time of day. Study medication was injected subcutaneously into the upper arm in the area of the deltoid or to the thigh. A maximum 600 mg dose required 4 injections. All participants who entered the study received itraconazole twice daily, while receiving oral corticosteroids, with a maximum daily dose of 400 mg. Participants who completed double-blinded phase, entered open-label treatment period of 6 months and continued the same regimen of omalizumab of double-blinded phase. | Drug: Omalizumab<br>* Omalizumab subcutaneous injections of 600 mg daily.<br>* Other names: IGE025;Drug: Itraconazole<br>* Itraconazole twice daily with a maximum daily dose of 400 mg.<br>* Other names: Sporanox;|
| Placebo Comparator: Placebo<br>Eligible participants received placebo comparator via subcutaneous injection for 6 months in the double-blind phase of the study. The study medication was to be administered at the same time of day. Study medication was injected subcutaneously into the upper arm in the area of the deltoid or to the thigh. All participants who entered the study received itraconazole twice daily, while on oral corticosteroids, with a maximum daily dose of 400 mg. | Drug: Placebo<br>* Placebo subcutaneous injections blinded to match experimental arm dosing regimen.<br>Drug: Itraconazole<br>* Itraconazole twice daily with a maximum daily dose of 400 mg.<br>* Other names: Sporanox;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline, as Measured by the Percentage of Participants Requiring Rescue With Corticosteroids, and as Measured by the Time to Deviation From the Protocol Prescribed Steroid Tapering Regimen |  | 6 months of blinded treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Allergic Bronchopulmonary Aspergillosis (ABPA) Exacerbation Rates During Double-blind Treatment Period and Open-label Treatment Period |  | 6 months, 12 months | 
| Change in Forced Expiratory Volume in 1 Second (FEV1) From Baseline, Measured at 3 and 6 Months of Treatment |  | 3 months, 6 months | 
| Time to Steroid Free State. |  | 12 months | 
| Change From Baseline in Average Oral Corticosteroid Use. |  | 6 months, 12 months | 
| Percentage of Participants Responding to Omalizumab, as Defined by a Reduction in Oral Corticosteroid Dose Use of 50% or More as Compared to Baseline |  | 6 months, 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis, Allergic Bronchopulmonary Aspergillosis, omalizumab, oral corticosteroid use, anti-immunoglobulin E