[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100592694":3},{"organization":4,"armGroups":7,"interventions":25,"overallOfficials":31,"centralContacts":35,"locations":45,"responsibleParty":64,"collaborators":66,"id":70,"slug":71,"hasResults":72,"nctId":73,"briefTitle":74,"officialTitle":74,"acronym":16,"eligibilityCriteria":75,"healthyVolunteers":72,"sex":76,"minAge":77,"maxAge":78,"enrollmentInfo":79,"targetDuration":16,"studyType":82,"phases":83,"briefSummary":85,"conditions":86,"keywords":88,"overallStatus":48,"whyStopped":16,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":98,"completionDateStruct":100,"leadSponsor":102,"locationsCount":103},{"fullName":5,"class":6},"Weill Medical College of Cornell University","OTHER",[8,14,18,21],{"label":9,"type":10,"description":11,"interventionNames":12},"AAV8hAAT(AVL) - 5x10¹¹ gc\u002Fkg","EXPERIMENTAL","Lowest dose of vector genome copies per kilogram",[13],"Biological: AAV8hAAT(AVL)",{"label":15,"type":10,"description":16,"interventionNames":17},"AAV8hAAT(AVL) - 2x10¹² gc\u002Fkg",null,[13],{"label":19,"type":10,"description":16,"interventionNames":20},"AAV8hAAT(AVL) - 5x10¹² gc\u002Fkg",[13],{"label":22,"type":10,"description":23,"interventionNames":24},"AAV8hAAT(AVL) - 2x10¹³ gc\u002Fkg","Highest dose of vector genome copies per kilogram",[13],[26],{"type":27,"name":28,"description":29,"armGroupLabels":30,"otherNames":16},"BIOLOGICAL","AAV8hAAT(AVL)","AAV8hAAT(AVL) gene transfer vector",[15,22,19,9],[32],{"name":33,"affiliation":5,"role":34},"Ronald G Crystal, MD","PRINCIPAL_INVESTIGATOR",[36,41],{"name":37,"role":38,"phone":39,"phoneExt":16,"email":40},"Niamh Savage","CONTACT","646-962-5527","nis2049@med.cornell.edu",{"name":42,"role":38,"phone":43,"phoneExt":16,"email":44},"Sandra Hyde","646-962-2672","sah2003@med.cornell.edu",[46],{"facility":47,"status":48,"city":49,"state":49,"zip":50,"country":51,"countryCode":52,"cosmosGeoPoint":53,"geoPoint":58,"contacts":59},"WCMC Department of Genetic Medicine","RECRUITING","New York","10021","United States","US",{"type":54,"coordinates":55},"Point",[56,57],-74.00597,40.71427,{"lat":57,"lon":56},[60,61,62],{"name":42,"role":38,"phone":43,"phoneExt":16,"email":44},{"name":37,"role":38,"phone":39,"phoneExt":16,"email":40},{"name":63,"role":34,"phone":16,"phoneExt":16,"email":16},"Ronald Crystal, MD",{"type":65,"investigatorFullName":16,"investigatorTitle":16,"investigatorAffiliation":16,"oldNameTitle":16,"oldOrganization":16},"SPONSOR",[67],{"name":68,"class":69},"National Heart, Lung, and Blood Institute (NHLBI)","NIH","100592694","phase-1-gene-therapy-for-alpha-1--antitrypsin-deficiency-100592694",false,"NCT06996756","Gene Therapy for Alpha 1- Antitrypsin Deficiency","Inclusion Criteria:\n\n* AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \\\u003C11 μM\n* Emphysema as assessed by chest high resolution computational tomography (HRCT)\n* Lung function parameters consistent with mild to moderate loss of lung function and the presence of emphysema.\n* Troponin T within normal limits\n* Normal liver ultrasound and serum alpha fetoprotein\n* Normal kidney function\n* No contraindications to receiving corticosteroid immunosuppression\n\nExclusion Criteria:\n\n* Individuals receiving systemic corticosteroids or other immunosuppressive medications for pre-existing conditions.\n* Inability to tolerate immunosuppression with corticosteroids (e.g., uncontrolled diabetes)\n* Individuals with an immunodeficiency disease, or evidence of active infection of any type, including human immunodeficiency virus\n* Evidence of major central nervous system, major psychiatric, musculoskeletal or immune disorder\n* Prior history of myocardial infarction or cancer within the past 5 years (other than basal cell carcinoma of the skin)\n* Decompensated heart failure (NY4A class III-IV at time of baseline clinical assessment)\n* Abnormal ECG at screening with findings consistent with cardiac disease\n* Females who are currently pregnant or lactating\n* Any history of allergies to drugs used for bronchoscopy, including xylocaine, lidocaine, versed, valium, atropine, pilocarpine, isoproterenol, terbutaline, aminophylline, or any local anesthetic\n* Individuals receiving experimental medications or participating in another experimental protocol for at least 3 months prior to entry to the study\n* Use of oxygen supplementation\n* Risk for thromboembolic disease\n* History of significant cardiovascular disease, hypertension, prior myocardial infarction and\u002For cerebrovascular event\n* Individuals who are currently on beta-blockers, or other cardiac therapy related drugs\n* Prior history of hypersensitivity or anaphylaxis associated with the administration of any AAT product","ALL","18 Years","70 Years",{"count":80,"type":81},16,"ESTIMATED","INTERVENTIONAL",[84],"PHASE1","This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on a persistent basis. We hope to learn the safety\u002Ftoxicity and initial evidence of efficacy of intravenous delivery of this gene therapy to alpha 1-antitrypsin deficient individuals.",[87],"Alpha 1-Antitrypsin Deficiency",[89,90,91,92,93],"AAT","Emphysema","Gene therapy","DLCO","Augmentation therapy","2026-03-11",{"date":96,"type":97},"2026-03-13","ACTUAL",{"date":99,"type":97},"2025-02-26",{"date":101,"type":81},"2032-08-01",{"name":5,"class":6},1]