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This study looks at an experimental gene transfer method (the rAAV2-CB-hAAT gene vector) that aims to help the body produce the missing protein in patients with alpha 1-antitrypsin deficiency. Patients diagnosed with alpha 1-antitrypsin deficiency whose lung function was above a certain level were able to take part. Sponsored by the University of Massachusetts, Worcester, the study was conducted in Early Phase 1 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Diagnosed with AAT deficiency * Forced expiratory volume in one second (FEV1) greater than 24% of predicted value (post bronchodilator) * Willing to discontinue AAT protein replacement 4 weeks prior to study entry, and to resume 11 weeks after rAAV2-CB-hAAT has been administered * Willing to discontinue aspirin, aspirin-containing products, and other drugs that may alter platelet function 7 days prior to study entry, and to resume 24 hours after rAAV2-CB-hAAT has been administered * Willing to use contraception throughout the study Exclusion Criteria: * Required antibiotic therapy for a respiratory infection in the 28 days prior to rAAV2-CB-hAAT administration * Required oral or systemic corticosteroids in the 28 days prior to rAAV2-CB-hAAT administration * Liver disease * Currently receiving or has received an investigational study agent in the 30 days prior to study entry * Received gene transfer agents in the 6 months prior to study entry * Currently smokes cigarettes or uses illegal drugs * History of immune response to human AAT replacement * History of platelet dysfunction * Any other medical condition that the investigator deems unsuitable for study participation * Pregnant or breastfeeding