Inhaled alpha-1 antitrypsin (AAT) restores lower respiratory tract protease-antiprotease homoeostasis and reduces inflammation in AAT-deficient individuals: a randomised phase 2 study.

ABSTRACT:

Background

Alpha-1 antitrypsin (AAT)-deficient individuals have a greater risk for developing COPD than individuals with normal AAT levels.

Methods

This was a double-blind, randomised, parallel group, placebo-controlled trial to examine the safety and tolerability of "Kamada-AAT for Inhalation" (inhaled AAT) in subjects with AAT deficiency, and to explore its effect on AAT and biomarkers in the lung epithelial lining fluid (ELF). 36 patients with severe AAT deficiency were randomised 2:1 to receive 80 mg or 160 mg inhaled AAT or placebo once daily for 12 weeks. The primary outcomes were AAT and antineutrophil elastase capacity (ANEC) in bronchoalveolar lavage and plasma after treatment. Secondary outcomes included safety, levels of normal M-type AAT in the plasma and concent

SUBMITTER: Brantly M 

PROVIDER: S-EPMC11726588 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature