Alpha1 Antitrypsin Aerosol Therapy in Cystic Fibrosis

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

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

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.

Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy.

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: -

McElvaney NG, Hubbard RC, Birrer P, Chernick MS, Caplan DB, Frank MM, Crystal RG. Aerosol alpha 1-antitrypsin treatment for cystic fibrosis. Lancet. 1991 Feb 16;337(8738):392-4. doi: 10.1016/0140-6736(91)91167-s.