Alpha1 Antitrypsin Aerosol Therapy in Cystic Fibrosis

Phase 2

Terminated

• Conditions: Cystic Fibrosis
• Interventions: Drug: Prolastin (drug)

• Registration Number: NCT02010411
• Lead Sponsor: Université de Sherbrooke

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.

Study Timeline

• Study Start: 2004-06
• Primary Completion: 2004-10
• Study Completion: 2005-10
• Status Verified: 2013-12
• Study First Submitted: 2013-12-09
• Study First Submitted QC: 2013-12-11
• Last Update Submitted: 2013-12-11
• Study First Posted: 2013-12-12
• Last Update Posted: 2013-12-12

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 17

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Prolastin	Prolastin (drug)	Prolastin 250 mg nebulized BID, 10 days

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Neutrophil burden in airways as determined by sputum myeloperoxidase;	6 hours after Prolastin
Alpha1 antitrypsin in sputum	6 hours after Prolastin
Sputum elastase activity	6 hours after Prolastin

Trial Locations

Locations (1)

Centre de Recherche du CHUS; 🇨🇦 Sherbrooke, Quebec, Canada