Clinical trial NCT02010411: Alpha1 Antitrypsin Aerosol Therapy in Cystic Fibrosis
Official title: Effects of Prolastin Aerosol Therapy on Bacterial Density in the Airways of Patients With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Cystic Fibrosis
Lead sponsor: Université de Sherbrooke
Sponsor class: OTHER
First posted: 2013-12-12
Enrollment: 17
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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:

-

Primary outcomes:
measure: Bacterial density in sputum as determined by colony forming units at 2, 4 and 6 hours after Prolastin therapy.
timeFrame: 6 hours after Prolastin
Secondary outcomes:
measure: Neutrophil burden in airways as determined by sputum myeloperoxidase;
timeFrame: 6 hours after Prolastin
measure: Sputum elastase activity
timeFrame: 6 hours after Prolastin
measure: Alpha1 antitrypsin in sputum
timeFrame: 6 hours after Prolastin
Other outcomes:
Locations:
city: Sherbrooke
country: Canada
facility: Centre de Recherche du CHUS
state: Quebec
Study officials:
affiliation: Centre de recherche du Centre hospitalier universitaire de Sherbrooke
name: Andre Cantin, md
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.