[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100610409":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":11,"centralContacts":20,"locations":26,"responsibleParty":60,"collaborators":11,"id":62,"slug":63,"hasResults":64,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":11,"eligibilityCriteria":68,"healthyVolunteers":64,"sex":69,"minAge":70,"maxAge":71,"enrollmentInfo":72,"targetDuration":11,"studyType":75,"phases":76,"briefSummary":79,"conditions":80,"keywords":11,"overallStatus":29,"whyStopped":11,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":91},{"fullName":5,"class":6},"Tessera Therapeutics, Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"TSRA-196 Drug Product","EXPERIMENTAL",null,[13],"Drug: TSRA-196",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":11},"DRUG","TSRA-196","TSRA-196 is an in-vivo genome editing product formulated in lipid nanoparticles (LNPs) for the treatment of patients with alpha-1 antitrypsin deficiency (AATD), via intravenous (IV) infusion",[9],[21],{"name":22,"role":23,"phone":24,"phoneExt":11,"email":25},"Tessera Clinical Trials Information","CONTACT","857-271-4800","clinicalinfo@tesseratx.com",[27,41,50],{"facility":28,"status":29,"city":30,"state":31,"zip":32,"country":33,"countryCode":34,"cosmosGeoPoint":35,"geoPoint":40,"contacts":11},"Clinical Study Center","RECRUITING","Boston","Massachusetts","02118","United States","US",{"type":36,"coordinates":37},"Point",[38,39],-71.05977,42.35843,{"lat":39,"lon":38},{"facility":28,"status":29,"city":42,"state":43,"zip":44,"country":33,"countryCode":34,"cosmosGeoPoint":45,"geoPoint":49,"contacts":11},"Charleston","South Carolina","29425",{"type":36,"coordinates":46},[47,48],-79.93275,32.77632,{"lat":48,"lon":47},{"facility":28,"status":29,"city":51,"state":52,"zip":11,"country":53,"countryCode":54,"cosmosGeoPoint":55,"geoPoint":59,"contacts":11},"Fitzroy","Victoria","Australia","AU",{"type":36,"coordinates":56},[57,58],144.97833,-37.79839,{"lat":58,"lon":57},{"type":61,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100610409","phase-1-a-study-of-tsra-196-in-adults-with-pizz-alpha-1-antitrypsin-deficiency-aatd-100610409",false,"NCT07227207","A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)","A Phase 1\u002F2, Open-Label, Multi-Center, Dose Escalation, Dose Expansion, and Single Repeat Dose Study of TSRA-196 in Adults With the PiZZ Genotype Who Have Lung and\u002For Liver Disease Associated With Severe Alpha-1 Antitrypsin Deficiency","Inclusion Criteria:\n\n* Males or females who are 18 to 70 years of age, inclusive, at the time of signing the informed consent\n* Body mass index of 18 to 37 kg\u002Fm2, inclusive\n* Confirmed diagnosis of AATD and PiZZ genotype\n* At least one previous measure of blood total AAT level \\\u003C11 µmol\u002FL\n* Nonsmoker for at least 6 months before screening and must remain nonsmoking for the entire study duration\n* Either AAT treatment-naïve or washed out of all investigational or approved treatments that modify AAT levels for 5 half-lives or at least 4 weeks, whichever is longer, before TSRA-196 administration\n\nParts 1A and 2A (AATD lung disease with no or minimal liver fibrosis)\n\n* Clinically significant lung disease, defined as 1) evidence of emphysema or bronchiectasis by computed tomography or 2) DLCO \\\u003C70% of the predicted value or 3) ppFEV1 \\\u003C80%\n* ppFEV1 ≥35%\n* METAVIR fibrosis score F0 or F1 confirmed by liver biopsy at screening, or a liver stiffness measure by FibroScan ≤7 kPa at screening\n* FIB-4 index score ≤3.25 at screening\n* ALT and\u002For AST \\\u003CULN at screening\n\nParts 1B and 2B (AATD liver disease with significant or severe liver fibrosis, with or without AATD lung disease)\n\n* METAVIR fibrosis score F2 or F3 confirmed by liver biopsy at screening. A liver biopsy conducted within 12 months before screening is acceptable as a substitute.\n* Liver stiffness measure by FibroScan \\>7 and ≤15 kPa at screening\n* ALT and\u002For AST \\\u003C2 x ULN at screening\n\nExclusion Criteria:\n\n* Presence of genetic variation in SERPINA1 gene that may disrupt the function of TSRA-196, determined by screening genotyping\n* History of liver disease unrelated to AATD, or history of or clinical signs of cirrhosis\n* Significant lung disease not attributable to manifestations of AATD, as determined by the investigator\n* History of one or more hospitalizations due to severe exacerbation of underlying lung disease during the year before screening or received IV antibiotics for treatment of a pulmonary infection within 6 months before screening\n* Unstable AATD-related COPD, as determined by the investigator, or severe bronchiectasis\n* Lung volume reduction surgery within 1 year before screening or plan to receive lung volume reduction surgery during the study period\n* Documented chronic need for positive airway pressure therapy beyond nocturnal use\n* Seropositive for human immunodeficiency virus (HIV) (HIV-1 or HIV-2)\n* Seropositive for hepatitis B (hepatitis B surface antigen \\[HBsAg\\] or hepatitis B core antibody \\[HBcAb\\] positive) with detectable HBV DNA\n* Hepatitis C virus (HCV) RNA positive at screening (Parts 1A and 2A), or HCV RNA positive and\u002For HCV antibody positive at screening (Parts 1B and 2B)\n* Has received an organ transplant or is on a waiting list for an organ transplant\n* Prior treatment with gene therapy using viral vectors or intended to permanently change the patient's DNA\n* Any investigational products within 30 days before dosing or plan to take an investigational product before the end of study","ALL","18 Years","70 Years",{"count":73,"type":74},72,"ESTIMATED","INTERVENTIONAL",[77,78],"PHASE1","PHASE2","This is a Phase 1\u002F2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK\u002FPD parameters of TSRA-196 in adults with the PiZZ genotype who have lung and\u002For liver disease associated with severe alpha-1 antitrypsin deficiency (AATD)",[81],"Alpha-1 Antitrypsin Deficiency (AATD)","2026-05-13",{"date":84,"type":85},"2026-05-18","ACTUAL",{"date":87,"type":85},"2026-04-21",{"date":89,"type":74},"2029-03",{"name":5,"class":6},3]