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This study investigates a gene therapy called AAV8hAAT(AVL) in patients with alpha-1 antitrypsin deficiency. It enrolls patients with a ZZ or Z null genotype who have emphysema in the lungs and mild to moderate loss of lung function. It is in Phase 1 and is currently recruiting.
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: * AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \<11 μM * Emphysema as assessed by chest high resolution computational tomography (HRCT) * Lung function parameters consistent with mild to moderate loss of lung function and the presence of emphysema. * Troponin T within normal limits * Normal liver ultrasound and serum alpha fetoprotein * Normal kidney function * No contraindications to receiving corticosteroid immunosuppression Exclusion Criteria: * Individuals receiving systemic corticosteroids or other immunosuppressive medications for pre-existing conditions. * Inability to tolerate immunosuppression with corticosteroids (e.g., uncontrolled diabetes) * Individuals with an immunodeficiency disease, or evidence of active infection of any type, including human immunodeficiency virus * Evidence of major central nervous system, major psychiatric, musculoskeletal or immune disorder * Prior history of myocardial infarction or cancer within the past 5 years (other than basal cell carcinoma of the skin) * Decompensated heart failure (NY4A class III-IV at time of baseline clinical assessment) * Abnormal ECG at screening with findings consistent with cardiac disease * Females who are currently pregnant or lactating * Any history of allergies to drugs used for bronchoscopy, including xylocaine, lidocaine, versed, valium, atropine, pilocarpine, isoproterenol, terbutaline, aminophylline, or any local anesthetic * Individuals receiving experimental medications or participating in another experimental protocol for at least 3 months prior to entry to the study * Use of oxygen supplementation * Risk for thromboembolic disease * History of significant cardiovascular disease, hypertension, prior myocardial infarction and/or cerebrovascular event * Individuals who are currently on beta-blockers, or other cardiac therapy related drugs * Prior history of hypersensitivity or anaphylaxis associated with the administration of any AAT product