Prospective Phase 3 Multi-center 2-Year Placebo Controlled Double Blind Study to Evaluate the Efficacy and Safety of Kamada-AAT for Inhalation 80 mg/Day in Alpha-1 Antitrypsin Deficiency With Moderate and Severe Airflow Limitation Followed by a 2-Year Open Label Extension

What this Trial Is About

This research aims to find out if inhaling Alpha 1-Antitrypsin (AAT) daily at a dose of 80 mg can slow down lung disease progression in people with lung problems caused by severe genetic Alpha 1 Antitrypsin Deficiency (AATD). The study is a Phase 3 trial comparing the inhaled AAT treatment to a placebo. It evaluates lung function using tests such as spirometry and CT scans to measure lung density and overall lung health. Participants will inhale either the study drug Kamada AAT or a placebo solution every day for two years during the double-blind phase. After this, all participants receive the active AAT treatment for an additional two years in an open-label extension. The treatment is given using a nebulizer device, and participants are asked to maintain daily cleaning of the device and record symptoms and drug use in an electronic diary. Throughout the study, participants will attend clinic visits: 11 visits during the first two years and 5-6 visits during the extension phase. After treatment ends, there will be three follow-up clinic visits over six months. Researchers will perform lung function tests, CT scans, six-minute walk tests, quality of life questionnaires, and biomarker assessments. The primary outcome measured is lung function (FEV1) after bronchodilator use at 104 weeks to understand the treatment's impact on lung disease progression.

Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"