Alpha1 Antitrypsin Aerosol Therapy in Cystic Fibrosis

Study Overview
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Brief Summary
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The hypothesis being tested is that inhibition of the enzyme known as elastase in the airways of patients with cystic fibrosis will help decrease the number of bacteria. Alpha1 antitrypsin, an elastase inhibitor, will be given to patients with cystic fibrosis by aerosol therapy twice in 1 day and sputum will be collected to measure the density of bacteria

Detailed Description
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Cystic fibrosis is usually characterized by chronic bacterial infections of the airways. Neutrophils release the enzyme elastase in the airways and this enzyme can prevent the ingestion and killing of bacteria by the airway phagocytic cells. The hypothesis being tested is that inhibition of elastase in the airways will help neutrophils decrease the number of bacteria. Each subject with cystic fibrosis will first undergo aerosol therapy with a sterile saline solution and sputum will be collected 2, 4 and 6 hours after the aerosol therapy to measure the density of bacteria. Subsequently, alpha1 antitrypsin, an elastase inhibitor, will be given to the same patients by aerosol therapy twice in 1 day and sputum will be collected at 2, 4 and 6 hours after treatment to measure the density of bacteria. The results will be compared to those obtained after after aerosol therapy with saline solution. Study phase II Study type Interventional Study design Purpose - treatment Allocation - nonrandomized trial Masking - open Control - active Assignment - cross-over Endpoint - efficacy Primary Outcome Measure Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy.

Official Title
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Effects of Prolastin Aerosol Therapy on Bacterial Density in the Airways of Patients With Cystic Fibrosis

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: Prolastin (drug)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of cystic fibrosis Age 14 years and older Women must have a negative pregnancy test and used effective contraception Must be able to produce sputum Sputum culture positive for Pseudomonas aeruginosa Exclusion Criteria: -

Ages Eligible for Study
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Minimum Age: 14 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Prolastin<br>Prolastin 250 mg nebulized BID, 10 days | Drug: Prolastin (drug)<br>* Outcomes compared to saline or no intervention in each subject.<br>* Other names: Alpha1 antitrypsin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy. |  | 6 hours after Prolastin | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Neutrophil burden in airways as determined by sputum myeloperoxidase; |  | 6 hours after Prolastin | 
| Sputum elastase activity |  | 6 hours after Prolastin | 
| Alpha1 antitrypsin in sputum |  | 6 hours after Prolastin |