[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100555466":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":24,"centralContacts":28,"locations":34,"responsibleParty":165,"collaborators":10,"id":167,"slug":168,"hasResults":169,"nctId":170,"briefTitle":171,"officialTitle":172,"acronym":173,"eligibilityCriteria":174,"healthyVolunteers":169,"sex":175,"minAge":176,"maxAge":10,"enrollmentInfo":177,"targetDuration":10,"studyType":180,"phases":10,"briefSummary":181,"conditions":182,"keywords":184,"overallStatus":37,"whyStopped":10,"lastUpdateSubmitDate":186,"lastUpdatePostDateStruct":187,"startDateStruct":190,"completionDateStruct":192,"leadSponsor":194,"locationsCount":195},{"fullName":5,"class":6},"Takeda","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort 1: AATD-Pi*ZZ Genotype\u002FPhenotype",null,"Participants who have been diagnosed with Alpha-1 Antitrypsin Deficiency homozygous ZZ (AATD-Pi\\*ZZ) genotype\u002Fphenotype with or without liver disease manifestations (fibrosis- F0-F4dc) will be enrolled and data will be prospectively collected per routine care throughout the follow-up period.",[13],"Other: No Intervention",{"label":15,"type":10,"description":16,"interventionNames":17},"Cohort 2: AATD-Pi*SZ Genotype\u002FPhenotype","Participants who have been diagnosed with alpha-1 antitrypsin deficiency heterozygous SZ (AATD-Pi\\*SZ) genotype\u002Fphenotype with moderate-advanced or severe liver disease (F2-F4dc) manifestations will be enrolled and data will be prospectively collected per routine care throughout the follow-up period.",[13],[19],{"type":20,"name":21,"description":22,"armGroupLabels":23,"otherNames":10},"OTHER","No Intervention","This is an observational study.",[9,15],[25],{"name":26,"affiliation":5,"role":27},"Study Director","STUDY_DIRECTOR",[29],{"name":30,"role":31,"phone":32,"phoneExt":10,"email":33},"Takeda Contact","CONTACT","+1-877-825-3327","medinfoUS@takeda.com",[35,56,71,86,102,118,134,149],{"facility":36,"status":37,"city":38,"state":39,"zip":40,"country":41,"countryCode":42,"cosmosGeoPoint":43,"geoPoint":48,"contacts":49},"University of Florida","RECRUITING","Gainesville","Florida","32608","United States","US",{"type":44,"coordinates":45},"Point",[46,47],-82.32483,29.65163,{"lat":47,"lon":46},[50,53],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":52},"Site Contact","Virginia.Clark@medicine.ufl.edu",{"name":54,"role":55,"phone":10,"phoneExt":10,"email":10},"Virginia Clark","PRINCIPAL_INVESTIGATOR",{"facility":57,"status":37,"city":58,"state":59,"zip":60,"country":41,"countryCode":42,"cosmosGeoPoint":61,"geoPoint":65,"contacts":66},"University of South Carolina","Charleston","South Carolina","29425",{"type":44,"coordinates":62},[63,64],-79.93275,32.77632,{"lat":64,"lon":63},[67,69],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":68},"strangec@musc.edu",{"name":70,"role":55,"phone":10,"phoneExt":10,"email":10},"Charlie Strange",{"facility":72,"status":37,"city":73,"state":74,"zip":75,"country":41,"countryCode":42,"cosmosGeoPoint":76,"geoPoint":80,"contacts":81},"Vanderbilt University Medical Center","Nashville","Tennessee","37212",{"type":44,"coordinates":77},[78,79],-86.78444,36.16589,{"lat":79,"lon":78},[82,84],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":83},"suzanne.sharpton@vumc.org",{"name":85,"role":55,"phone":10,"phoneExt":10,"email":10},"Suzanne Sharpton",{"facility":87,"status":37,"city":88,"state":10,"zip":89,"country":90,"countryCode":91,"cosmosGeoPoint":92,"geoPoint":96,"contacts":97},"Vienna General Hospital (AKH Wien)","Vienna","1090","Austria","AT",{"type":44,"coordinates":93},[94,95],16.37208,48.20849,{"lat":95,"lon":94},[98,100],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":99},"mattias.mandorfer@meduniwien.ac.at",{"name":101,"role":55,"phone":10,"phoneExt":10,"email":10},"Mattias Mandorfer",{"facility":103,"status":37,"city":104,"state":10,"zip":105,"country":106,"countryCode":107,"cosmosGeoPoint":108,"geoPoint":112,"contacts":113},"Universitätsklinikum Aachen","Aachen","52074","Germany","DE",{"type":44,"coordinates":109},[110,111],6.08342,50.77664,{"lat":111,"lon":110},[114,116],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":115},"pstrnad@ukaachen.de",{"name":117,"role":55,"phone":10,"phoneExt":10,"email":10},"Pavel Strnad",{"facility":119,"status":37,"city":120,"state":10,"zip":121,"country":122,"countryCode":123,"cosmosGeoPoint":124,"geoPoint":128,"contacts":129},"Beaumont Hospital","Dublin","Dublin 9","Ireland","IE",{"type":44,"coordinates":125},[126,127],-6.24889,53.33306,{"lat":127,"lon":126},[130,132],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":131},"gmcelvaney@rcsi.ie",{"name":133,"role":55,"phone":10,"phoneExt":10,"email":10},"Gerry McElvaney",{"facility":135,"status":37,"city":136,"state":10,"zip":10,"country":137,"countryCode":138,"cosmosGeoPoint":139,"geoPoint":143,"contacts":144},"Hospital Universitari Vall d'Hebron","Barcelona","Spain","ES",{"type":44,"coordinates":140},[141,142],2.15899,41.38879,{"lat":142,"lon":141},[145,147],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":146},"monica.pons@vhir.org",{"name":148,"role":55,"phone":10,"phoneExt":10,"email":10},"Monica Pons Delgado",{"facility":150,"status":37,"city":151,"state":10,"zip":152,"country":153,"countryCode":154,"cosmosGeoPoint":155,"geoPoint":159,"contacts":160},"Queen Elizabeth Hospital Birmingham","Birmingham","B15 2GW","United Kingdom","UK",{"type":44,"coordinates":156},[157,158],-1.89983,52.48142,{"lat":158,"lon":157},[161,163],{"name":51,"role":31,"phone":10,"phoneExt":10,"email":162},"a.m.turner@bham.ac.uk",{"name":164,"role":55,"phone":10,"phoneExt":10,"email":10},"Alice Turner",{"type":166,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR","100555466","a-study-in-adults-to-learn-about-inherited-alpha-1-antitrypsin-deficiency-aatd-and-aatd-related-liver-problems-100555466",false,"NCT06512454","A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems","Prospective Observational Study on the Natural History of Alpha-1 Antitrypsin Deficiency and Associated Liver Disease","ALPHATUDE","Inclusion Criteria:\n\nParticipants who meet all the following criteria will be included in the study.\n\nCohorts 1 and 2:\n\n1. Willing to provide written informed consent to participate in the study.\n2. \\>=18 years of age at enrollment in this study.\n3. Participants with documented diagnosis of AATD, meeting the following criteria:\n\n   1. Cohort 1 (AATD-Pi\\*ZZ genotype\u002Fphenotype).\n\n      • Pi\\*ZZ genotype as documented from rapid genetic assay, sequencing, or polymerase chain reaction (PCR), or Pi\\*ZZ phenotype as documented from iso-electric focusing (IEF) electrophoresis.\n   2. Cohort 2 (AATD-Pi\\*SZ genotype\u002Fphenotype with liver disease manifestation).\n\n      * Pi\\*SZ genotype as documented from rapid genetic assay, sequencing, or PCR, or Pi\\*SZ phenotype as documented from IEF electrophoresis, and\n      * Moderate-advanced or severe liver disease manifestation as defined by either liver biopsy or surrogate laboratory or imaging measures.\n\nExclusion Criteria:\n\nParticipants who meet any following criteria will be excluded from the study.\n\n1. Documented AATD genotype\u002Fphenotype other than Pi\\*ZZ or Pi\\*SZ.\n2. History of liver transplant.\n3. No results for either biopsies, magnetic resonance elastography (MRE), FibroScan (vibration controlled transient elastography \\[VCTE\\]), or Aspartate aminotransferase to platelet ratio index (APRI) in the 24 months prior to the index\u002Fenrollment date and has none of these tests ordered during the index period (i.e., index date +90 days).\n4. Participants with prior participation in an interventional clinical trial evaluating liver or lung disease, or who have received an investigational AATD-directed therapy under a compassionate use program, will be excluded if they do not present one of the following:\n\n   * A minimum washout period of 6 months has elapsed since the last dose of the investigational product.\n   * A history of having received placebo in prior interventional trials (to be evaluated on a case-by-case basis).","ALL","18 Years",{"count":178,"type":179},500,"ESTIMATED","OBSERVATIONAL","The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of AAT, called Z-AAT. Z-AAT builds up in liver cells and also leads to low blood levels of AAT (called Alpha-1 Antitrypsin Deficiency or AATD). Over time, this build up leads to different stages of liver problems, if not treated. This is called natural history of AATD.\n\nThe main aim of this study is to learn about liver problems caused by AATD in adults when not treated over 4 to 8 years. Other aims are to learn what can predict the AATD-liver condition starting and getting better or worse, describe how this condition is currently being diagnosed and watched in normal care, and describe how the AATD also affects an adult's lung function.\n\nData in this study will be collected to include medical history of a participant, including the date AATD was first identified and\u002For the date on which the first AATD-related liver or lung problems were diagnosed. At study start and then every year until study end, participants will be asked to complete questionnaires (called patient-reported outcomes or PROs).",[183],"Alpha1-Antitrypsin Deficiency",[185],"Natural History","2026-07-23",{"date":188,"type":189},"2026-07-24","ACTUAL",{"date":191,"type":189},"2024-09-25",{"date":193,"type":179},"2031-12-31",{"name":5,"class":6},8]