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

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.

Timeline

Start

2009-12

Primary completion

2014-11

Completion

2015-06

Outcome

Missed primary endpoint

paper AAT inhalation for 50 weeks showed no effect on time to first exacerbation PMID 31467115 ↗

release “study's primary endpoint of “Time to the first moderate or severe exacerbation event” did not show a statistically significant difference between inhaled AAT and placebo” kamada.com ↗

Publications

• Results Stolk J, Tov N, Chapman KR, Fernandez P, MacNee W, Hopkinson NS, Piitulainen E, Seersholm N, Vogelmeier CF, Bals R, McElvaney G, Stockley RA. Efficacy and safety of inhaled alpha1-antitrypsin in patients with severe alpha1-antitrypsin deficiency and frequent exacerbations of COPD. Eur Respir J. 2019 Nov 21;54(5):1900673. doi: 10.1183/13993003.00673-2019. Print 2019 Nov.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Alpha 1-Antitrypsin	Protein / enzyme biologic	80 mg	Inhaled

Indications