[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100546049":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":25,"locations":30,"responsibleParty":129,"collaborators":11,"id":131,"slug":132,"hasResults":133,"nctId":134,"briefTitle":135,"officialTitle":136,"acronym":11,"eligibilityCriteria":137,"healthyVolunteers":133,"sex":138,"minAge":139,"maxAge":140,"enrollmentInfo":141,"targetDuration":11,"studyType":144,"phases":145,"briefSummary":148,"conditions":149,"keywords":11,"overallStatus":33,"whyStopped":11,"lastUpdateSubmitDate":151,"lastUpdatePostDateStruct":152,"startDateStruct":155,"completionDateStruct":157,"leadSponsor":159,"locationsCount":160},{"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",[31,45,54,63,72,79,88,97,106,113,122],{"facility":32,"status":33,"city":34,"state":35,"zip":36,"country":37,"countryCode":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":11},"Clinical Study Center","RECRUITING","Birmingham","Alabama","35233","United States","US",{"type":40,"coordinates":41},"Point",[42,43],-86.80249,33.52066,{"lat":43,"lon":42},{"facility":32,"status":33,"city":46,"state":47,"zip":48,"country":37,"countryCode":38,"cosmosGeoPoint":49,"geoPoint":53,"contacts":11},"Boston","Massachusetts","02215",{"type":40,"coordinates":50},[51,52],-71.05977,42.35843,{"lat":52,"lon":51},{"facility":32,"status":33,"city":55,"state":56,"zip":57,"country":37,"countryCode":38,"cosmosGeoPoint":58,"geoPoint":62,"contacts":11},"Charleston","South Carolina","29425",{"type":40,"coordinates":59},[60,61],-79.93275,32.77632,{"lat":61,"lon":60},{"facility":32,"status":33,"city":64,"state":11,"zip":11,"country":65,"countryCode":66,"cosmosGeoPoint":67,"geoPoint":71,"contacts":11},"Adelaide","Australia","AU",{"type":40,"coordinates":68},[69,70],138.59863,-34.92866,{"lat":70,"lon":69},{"facility":32,"status":33,"city":73,"state":11,"zip":11,"country":65,"countryCode":66,"cosmosGeoPoint":74,"geoPoint":78,"contacts":11},"Fitzroy",{"type":40,"coordinates":75},[76,77],144.97833,-37.79839,{"lat":77,"lon":76},{"facility":32,"status":33,"city":80,"state":11,"zip":11,"country":81,"countryCode":82,"cosmosGeoPoint":83,"geoPoint":87,"contacts":11},"Dublin","Ireland","IE",{"type":40,"coordinates":84},[85,86],-6.24889,53.33306,{"lat":86,"lon":85},{"facility":32,"status":33,"city":89,"state":11,"zip":11,"country":90,"countryCode":91,"cosmosGeoPoint":92,"geoPoint":96,"contacts":11},"Leiden","Netherlands","NL",{"type":40,"coordinates":93},[94,95],4.49306,52.15833,{"lat":95,"lon":94},{"facility":32,"status":33,"city":98,"state":11,"zip":11,"country":99,"countryCode":100,"cosmosGeoPoint":101,"geoPoint":105,"contacts":11},"Auckland","New Zealand","NZ",{"type":40,"coordinates":102},[103,104],174.76349,-36.84853,{"lat":104,"lon":103},{"facility":32,"status":33,"city":107,"state":11,"zip":11,"country":99,"countryCode":100,"cosmosGeoPoint":108,"geoPoint":112,"contacts":11},"Hamilton",{"type":40,"coordinates":109},[110,111],175.28333,-37.78333,{"lat":111,"lon":110},{"facility":32,"status":33,"city":114,"state":11,"zip":11,"country":115,"countryCode":116,"cosmosGeoPoint":117,"geoPoint":121,"contacts":11},"London","United Kingdom","UK",{"type":40,"coordinates":118},[119,120],-0.12574,51.50853,{"lat":120,"lon":119},{"facility":32,"status":33,"city":123,"state":11,"zip":11,"country":115,"countryCode":116,"cosmosGeoPoint":124,"geoPoint":128,"contacts":11},"Southampton",{"type":40,"coordinates":125},[126,127],-1.40428,50.90395,{"lat":127,"lon":126},{"type":130,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100546049","phase-1-a-study-to-evaluate-the-safety-and-efficacy-of-beam-302-in-adult-patients-with-alpha-1-antitrypsin-deficiency-aatd-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":142,"type":143},106,"ESTIMATED","INTERVENTIONAL",[146,147],"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).",[150],"Alpha 1-Antitrypsin Deficiency","2026-03-18",{"date":153,"type":154},"2026-03-20","ACTUAL",{"date":156,"type":154},"2024-06-19",{"date":158,"type":143},"2030-05",{"name":5,"class":6},11]