Goal 2: Reduce Human Disease

Clinical trials of AAT augmentation therapy

Alpha-1 antitrypsin augmentation therapy is widely used for PI ZZ subjects, but randomized clinical trials of AAT augmentation therapy have not been adequately powered to assess the efficacy of this treatment. A clinical trial using COPD exacerbation frequency as the primary outcome, rather than decline in lung function, in PI ZZ subjects with moderate-to-severe COPD could provide definitive evidence for the utility of AAT augmentation therapy.

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Idea No. 1593