[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100546049":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":25,"locations":30,"responsibleParty":123,"collaborators":11,"id":125,"slug":11,"hasResults":126,"nctId":127,"briefTitle":128,"officialTitle":129,"acronym":11,"eligibilityCriteria":130,"healthyVolunteers":126,"sex":131,"minAge":132,"maxAge":133,"enrollmentInfo":134,"targetDuration":11,"studyType":137,"phases":138,"briefSummary":141,"conditions":142,"keywords":11,"overallStatus":33,"whyStopped":11,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":148,"completionDateStruct":150,"leadSponsor":152,"locationsCount":153},{"fullName":5,"class":6},"Beam Therapeutics Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"BEAM-302 Drug Product","EXPERIMENTAL",null,[13],"Drug: BEAM-302",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":11},"DRUG","BEAM-302","BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD",[9],[21],{"name":22,"affiliation":23,"role":24},"Medical Information","Beam Therapeutics","STUDY_DIRECTOR",[26],{"name":22,"role":27,"phone":28,"phoneExt":11,"email":29},"CONTACT","857-327-8641","clinicalinfo@beamtx.com",[31,44,53,62,70,77,85,93,101,108,116],{"facility":32,"status":33,"city":34,"state":35,"zip":36,"country":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":11},"Clinical Study Center","RECRUITING","Birmingham","Alabama","35233","United States",{"type":39,"coordinates":40},"Point",[41,42],-86.80249,33.52066,{"lat":42,"lon":41},{"facility":32,"status":33,"city":45,"state":46,"zip":47,"country":37,"cosmosGeoPoint":48,"geoPoint":52,"contacts":11},"Boston","Massachusetts","02215",{"type":39,"coordinates":49},[50,51],-71.05977,42.35843,{"lat":51,"lon":50},{"facility":32,"status":33,"city":54,"state":55,"zip":56,"country":37,"cosmosGeoPoint":57,"geoPoint":61,"contacts":11},"Charleston","South Carolina","29425",{"type":39,"coordinates":58},[59,60],-79.93275,32.77632,{"lat":60,"lon":59},{"facility":32,"status":33,"city":63,"state":11,"zip":11,"country":64,"cosmosGeoPoint":65,"geoPoint":69,"contacts":11},"Adelaide","Australia",{"type":39,"coordinates":66},[67,68],138.59863,-34.92866,{"lat":68,"lon":67},{"facility":32,"status":33,"city":71,"state":11,"zip":11,"country":64,"cosmosGeoPoint":72,"geoPoint":76,"contacts":11},"Fitzroy",{"type":39,"coordinates":73},[74,75],144.97833,-37.79839,{"lat":75,"lon":74},{"facility":32,"status":33,"city":78,"state":11,"zip":11,"country":79,"cosmosGeoPoint":80,"geoPoint":84,"contacts":11},"Dublin","Ireland",{"type":39,"coordinates":81},[82,83],-6.24889,53.33306,{"lat":83,"lon":82},{"facility":32,"status":33,"city":86,"state":11,"zip":11,"country":87,"cosmosGeoPoint":88,"geoPoint":92,"contacts":11},"Leiden","Netherlands",{"type":39,"coordinates":89},[90,91],4.49306,52.15833,{"lat":91,"lon":90},{"facility":32,"status":33,"city":94,"state":11,"zip":11,"country":95,"cosmosGeoPoint":96,"geoPoint":100,"contacts":11},"Auckland","New Zealand",{"type":39,"coordinates":97},[98,99],174.76349,-36.84853,{"lat":99,"lon":98},{"facility":32,"status":33,"city":102,"state":11,"zip":11,"country":95,"cosmosGeoPoint":103,"geoPoint":107,"contacts":11},"Hamilton",{"type":39,"coordinates":104},[105,106],175.28333,-37.78333,{"lat":106,"lon":105},{"facility":32,"status":33,"city":109,"state":11,"zip":11,"country":110,"cosmosGeoPoint":111,"geoPoint":115,"contacts":11},"London","United Kingdom",{"type":39,"coordinates":112},[113,114],-0.12574,51.50853,{"lat":114,"lon":113},{"facility":32,"status":33,"city":117,"state":11,"zip":11,"country":110,"cosmosGeoPoint":118,"geoPoint":122,"contacts":11},"Southampton",{"type":39,"coordinates":119},[120,121],-1.40428,50.90395,{"lat":121,"lon":120},{"type":124,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100546049",false,"NCT06389877","A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)","A Phase 1\u002F2 Dose-exploration and Dose-expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and\u002For Liver Disease","Part A:\n\nInclusion Criteria:\n\n* Males or females 18 - 70 years of age inclusive at the time of consent.\n* Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).\n* Blood total AAT level \\\u003C11 μM or equivalent protein in mg\u002FdL.\n* Patients receiving augmentation therapy in regions where augmentation is not SoC must be willing to washout augmentation therapy for at least 6 weeks prior to signing the ICF and for the length of the study (unless clinically indicated)\n* A postbronchodilator FEV1 ≥40% of predicted and an FEV1\u002FFVC \\\u003C70% at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n* Evidence of emphysema on a historic CT scan or a DLCO ≤70% of the predicted value (corrected for hemoglobin) at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n\nExclusion Criteria:\n\n* Body mass index \\>30\n* Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.\n* Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\]).\n* Liver disease with any of the following:\n\n  * FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI \\>0.5 can be used as a surrogate exclusion criterion \\[Yilmaz, 2011\\].\n  * Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).\n  * Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.\n  * Have ALT or AST \\> upper limit of normal (ULN).\n  * Total bilirubin levels \\> ULN; if documented Gilbert's Syndrome, total bilirubin \\>2 × ULN.\n  * INR ≥1.2 at screening. If deemed appropriate by the investigator and\u002For prescribing physician, the patient may stop taking anticoagulants for an appropriate washout period or reversal with vitamin K and if indicated, a repeat INR within \\\u003C1.2 would be acceptable.\n  * Seropositive for hepatitis B (positive surface Ag).\n  * Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative.\n\nPart B:\n\nInclusion Criteria:\n\n* Males or females 18 - 70 years of age inclusive at the time of consent.\n* Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).\n* Evidence of METAVIR F1, F2, or F3 liver fibrosis based on a central read of a baseline liver biopsy during the screening period or a histological diagnosis made no more than 6 months before enrollment and stage confirmed by central read.\n* A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n\nExclusion Criteria:\n\n* Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.\n* Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\])\n* Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).","ALL","18 Years","70 Years",{"count":135,"type":136},106,"ESTIMATED","INTERVENTIONAL",[139,140],"PHASE1","PHASE2","This is a Phase 1\u002F2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK\u002FPD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and\u002For liver disease and to determine the optimal biological dose (OBD).",[143],"Alpha 1-Antitrypsin Deficiency","2026-03-18",{"date":146,"type":147},"2026-03-20","ACTUAL",{"date":149,"type":147},"2024-06-19",{"date":151,"type":136},"2030-05",{"name":5,"class":6},11]