[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100610409":3},{"organization":4,"outcomesModule":7,"designInfo":48,"detailedDescription":11,"studyPopulation":11,"armGroups":54,"interventions":60,"overallOfficials":11,"centralContacts":66,"locations":72,"responsibleParty":106,"collaborators":11,"id":108,"slug":11,"hasResults":109,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":11,"eligibilityCriteria":113,"healthyVolunteers":109,"sex":114,"minAge":115,"maxAge":116,"enrollmentInfo":117,"targetDuration":11,"studyType":120,"phases":121,"briefSummary":124,"conditions":125,"keywords":11,"overallStatus":75,"whyStopped":11,"lastUpdateSubmitDate":127,"lastUpdatePostDateStruct":128,"startDateStruct":131,"completionDateStruct":133,"leadSponsor":135,"locationsCount":136},{"fullName":5,"class":6},"Tessera Therapeutics, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":19,"otherOutcomes":11},[9,13,15,17],{"measure":10,"description":11,"timeFrame":12},"Part 1 (Dose Escalation): Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)",null,"1 Year",{"measure":14,"description":11,"timeFrame":12},"Part 2 (Dose Expansion): Proportion of participants who have serum levels of total alpha-1 antitrypsin (AAT) greater than or equal to Lower Limit of Normal (LLN) after TSRA-196 treatment",{"measure":16,"description":11,"timeFrame":12},"Part 2 (Dose Expansion): Change in functional AAT concentrations (determined using an elastase inhibition assay) from baseline to end of study",{"measure":18,"description":11,"timeFrame":12},"Part 3 (Single Repeated Dose): Proportion of participants who have serum levels of total alpha-1 antitrypsin (AAT) greater than or equal to Lower Limit of Normal (LLN) after a second dose of TSRA-196",[20,22,24,26,28,30,32,34,36,38,40,42,44,46],{"measure":21,"description":11,"timeFrame":12},"Part 1 (Dose Escalation): Proportion of participants who have serum levels of total AAT greater than or equal to LLN after TSRA-196 treatment",{"measure":23,"description":11,"timeFrame":12},"Part 2 (Dose Expansion): Incidence of TEAEs and SAEs",{"measure":25,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): Proportion of participants who have serum levels of total AAT greater than or equal to 11 μM after TSRA-196 treatment",{"measure":27,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): Change in serum levels of total AAT from baseline over time",{"measure":29,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): Incidence of Adverse Event of Special Interest (AESI) from day of dosing through end of study",{"measure":31,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): Change over time in safety measures, including clinical laboratory parameters, vital signs, and ECG parameters",{"measure":33,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): Pharmacokinetic (PK) parameter: Area under the blood concentration time curves (AUC) of TSRA-196",{"measure":35,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): PK parameter: Maximum observed blood concentration (Cmax) of TSRA-196",{"measure":37,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): PK parameter: Time to Cmax (tmax) of TSRA-196",{"measure":39,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): PK parameter: Half-life (t1\u002F2) of TSRA-196",{"measure":41,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): PK parameter: Clearance (CL) of TSRA-196",{"measure":43,"description":11,"timeFrame":12},"Part 1 (Dose Escalation) and Part 2 (Dose Expansion): PK parameter: The volume of distribution at terminal stage (Vz) of TSRA-196",{"measure":45,"description":11,"timeFrame":12},"Part 2 (Dose Expansion): Change in post-bronchodilator percent predicted forced expiratory volume (ppFEV1) from baseline through end of study",{"measure":47,"description":11,"timeFrame":12},"Part 2 (Dose Expansion): Incidence of COPD exacerbations from baseline over time",{"allocation":49,"interventionModel":50,"interventionModelDescription":11,"primaryPurpose":51,"observationalModel":11,"timePerspective":11,"maskingInfo":52},"NA","SINGLE_GROUP","TREATMENT",{"masking":53,"maskingDescription":11,"whoMasked":11},"NONE",[55],{"label":56,"type":57,"description":11,"interventionNames":58},"TSRA-196 Drug Product","EXPERIMENTAL",[59],"Drug: TSRA-196",[61],{"type":62,"name":63,"description":64,"armGroupLabels":65,"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",[56],[67],{"name":68,"role":69,"phone":70,"phoneExt":11,"email":71},"Tessera Clinical Trials Information","CONTACT","857-271-4800","clinicalinfo@tesseratx.com",[73,87,96],{"facility":74,"status":75,"city":76,"state":77,"zip":78,"country":79,"countryCode":80,"cosmosGeoPoint":81,"geoPoint":86,"contacts":11},"Clinical Study Center","RECRUITING","Boston","Massachusetts","02118","United States","US",{"type":82,"coordinates":83},"Point",[84,85],-71.05977,42.35843,{"lat":85,"lon":84},{"facility":74,"status":75,"city":88,"state":89,"zip":90,"country":79,"countryCode":80,"cosmosGeoPoint":91,"geoPoint":95,"contacts":11},"Charleston","South Carolina","29425",{"type":82,"coordinates":92},[93,94],-79.93275,32.77632,{"lat":94,"lon":93},{"facility":74,"status":75,"city":97,"state":98,"zip":11,"country":99,"countryCode":100,"cosmosGeoPoint":101,"geoPoint":105,"contacts":11},"Fitzroy","Victoria","Australia","AU",{"type":82,"coordinates":102},[103,104],144.97833,-37.79839,{"lat":104,"lon":103},{"type":107,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","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":118,"type":119},72,"ESTIMATED","INTERVENTIONAL",[122,123],"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)",[126],"Alpha-1 Antitrypsin Deficiency (AATD)","2026-05-13",{"date":129,"type":130},"2026-05-18","ACTUAL",{"date":132,"type":130},"2026-04-21",{"date":134,"type":119},"2029-03",{"name":5,"class":6},3]