NCT01217671

International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients With Emphysema

A Phase II/III, Double-Blind, Randomized, Placebo-Controlled, Multicenter, International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients With Emphysema

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 168 (actual)
• Sponsor: Kamada, Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a randomised , placebo controlled, double blind , multicentre, Phase II/III study evaluating the safety and efficacy of Kamada AAT for inhalation in patients with Emphysema caused by Alpha-1 Antitrypsin (AAT) deficiency.

Detailed description

Alpha-1 Antitrypsin Deficiency, also called Alpha-1-Proteinase Inhibitor (API) deficiency, is a genetic disorder characterized by the production of an abnormal amount of AAT protein and reduced circulating levels of this protein. Subjects with AAT deficiency are at increased risk for developing Emphysema. It is believed that this is the result of the chronic activity of elastase released by cells continually present in the lungs in low numbers. Three blinded interim analyses have shown that there are no safety issues and no concerns regarding tolerability.

Conditions

• Emphysema

Interventions

Type	Name	Description
BIOLOGICAL	Kamada AAT for inhalation	Alpha-1 Antitrypsin (AAT)
OTHER	Placebo	Placebo

Timeline

• Start date: 2009-12-01
• Primary completion: 2014-11-01
• Completion: 2015-06-01
• First posted: 2010-10-08
• Last updated: 2019-11-29

Locations

12 sites across 7 countries: Canada, Denmark, Germany, Ireland, Netherlands, Sweden, United Kingdom