Clinical Trials

Alpha-1 Antitrypsin Deficiency

Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha 1 Antitrypsin (rAAV1-CB-hAAT) in Patients with Alpha 1 Antitrypsin Deficiency  PI: Terry Flotte MD  Sponsor: AGTC/NHLBI | Registration:  NCT01054339

This research study is in long term follow up. This study tested whether a study drug, called rAAV1-CB-hAAT, is safe and was designed to test what dose vector is needed to increase the amount of alpha-1 protein in the blood in affected individulas. The study drug contains a normal copy of the AAT gene and that is delivered via a rAAV1 through injections into the muscles.