[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06389877":3,"trial-entities:NCT06389877":164,"trial-summary:NCT06389877":170},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":21,"primary_purpose":22,"phases":23,"enrollment_info":26,"interventions":29,"primary_outcomes":36,"secondary_outcomes":45,"sex":50,"minimum_age":51,"maximum_age":52,"healthy_volunteers":53,"eligibility_criteria":54,"std_ages":80,"locations":83,"central_contacts":152,"overall_officials":158,"references":162,"see_also_links":163},"NCT06389877","BTX-302-001","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","RECRUITING","2030-05","2026-03","2026-03-20","2024-06-19","Beam Therapeutics Inc.","INDUSTRY",true,"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).",null,[19],"Alpha 1-Antitrypsin Deficiency",[],"INTERVENTIONAL","TREATMENT",[24,25],"PHASE1","PHASE2",{"count":27,"type":28},106,"ESTIMATED",[30],{"type":31,"name":32,"description":33,"armGroupLabels":34},"DRUG","BEAM-302","BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD",[35],"BEAM-302 Drug Product",[37,41],{"measure":38,"description":39,"timeFrame":40},"Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)","Numbers and percentages of patients reporting a given AE","2 years",{"measure":42,"description":43,"timeFrame":44},"Phase 2 Dose Expansion: Absolute blood levels of total AAT","Absolute Levels of AAT over time","2 Years",[46,48],{"measure":47,"description":43,"timeFrame":44},"Phase 1 Dose Exploration: Absolute blood levels of total AAT",{"measure":49,"description":39,"timeFrame":44},"Phase 2 Dose Expansion: Rates of TEAEs and SAEs","ALL","18 Years","70 Years",false,{"inclusion":55,"exclusion":64,"raw_text":79},[56,57,58,59,60,61,56,57,62,63],"Males or females 18 - 70 years of age inclusive at the time of consent.","Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).","Blood total AAT level \\\u003C11 μM or equivalent protein in mg\u002FdL.","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)","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.)","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.)","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.","A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)",[65,66,67,68,69,70,71,72,73,74,75,76,66,77,78],"Body mass index \\>30","Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.","Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\]).","Liver disease with any of the following:","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\\].","Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).","Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.","Have ALT or AST \\> upper limit of normal (ULN).","Total bilirubin levels \\> ULN; if documented Gilbert's Syndrome, total bilirubin \\>2 × ULN.","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.","Seropositive for hepatitis B (positive surface Ag).","Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative.","Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\])","Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).","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).",[81,82],"ADULT","OLDER_ADULT",[84,93,100,107,113,118,124,130,136,141,147],{"facility":85,"status":8,"city":86,"state":87,"zip":88,"country":89,"geoPoint":90},"Clinical Study Center","Birmingham","Alabama","35233","United States",{"lat":91,"lon":92},33.52066,-86.80249,{"facility":85,"status":8,"city":94,"state":95,"zip":96,"country":89,"geoPoint":97},"Boston","Massachusetts","02215",{"lat":98,"lon":99},42.35843,-71.05977,{"facility":85,"status":8,"city":101,"state":102,"zip":103,"country":89,"geoPoint":104},"Charleston","South Carolina","29425",{"lat":105,"lon":106},32.77632,-79.93275,{"facility":85,"status":8,"city":108,"country":109,"geoPoint":110},"Adelaide","Australia",{"lat":111,"lon":112},-34.92866,138.59863,{"facility":85,"status":8,"city":114,"country":109,"geoPoint":115},"Fitzroy",{"lat":116,"lon":117},-37.79839,144.97833,{"facility":85,"status":8,"city":119,"country":120,"geoPoint":121},"Dublin","Ireland",{"lat":122,"lon":123},53.33306,-6.24889,{"facility":85,"status":8,"city":125,"country":126,"geoPoint":127},"Leiden","Netherlands",{"lat":128,"lon":129},52.15833,4.49306,{"facility":85,"status":8,"city":131,"country":132,"geoPoint":133},"Auckland","New Zealand",{"lat":134,"lon":135},-36.84853,174.76349,{"facility":85,"status":8,"city":137,"country":132,"geoPoint":138},"Hamilton",{"lat":139,"lon":140},-37.78333,175.28333,{"facility":85,"status":8,"city":142,"country":143,"geoPoint":144},"London","United Kingdom",{"lat":145,"lon":146},51.50853,-0.12574,{"facility":85,"status":8,"city":148,"country":143,"geoPoint":149},"Southampton",{"lat":150,"lon":151},50.90395,-1.40428,[153],{"name":154,"role":155,"phone":156,"email":157},"Medical Information","CONTACT","857-327-8641","clinicalinfo@beamtx.com",[159],{"name":154,"affiliation":160,"role":161},"Beam Therapeutics","STUDY_DIRECTOR",[],[],{"nct_id":4,"conditions":165,"biomarkers":167},[166],"Alpha-1 Antitrypsin Deficiency",[168,169],"Aspartate Aminotransferase","PiZZ",{"nct_id":4,"found":15,"summary":171,"prompt_version":181},{"design":172,"status":173,"heading":174,"summary":175,"follow_up":176,"word_count":177,"commitments":178,"compensation":179,"drugs_mentioned":180},"This is a Phase 1\u002F2, open-label study, meaning all participants will receive BEAM-302 and know what they are getting. It plans to enroll 106 adult participants.","completed","BEAM-302 for Alpha-1 Antitrypsin Deficiency (AATD)","This study is testing a new treatment called BEAM-302 for adults with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition. BEAM-302 is a special type of therapy delivered using tiny fat particles. We want to see how safe and effective BEAM-302 is, and find the best dose. To join, you must be 18-70 years old, have AATD with the PiZZ mutation, and low levels of AAT in your blood. The study will measure side effects and how much AAT is in your blood over two years to see if the treatment is working. The current recruitment status is unclear.","Participants will be followed for up to 2 years to measure safety and AAT levels.",98,"Not specified in the trial record.","Not stated in the trial record.",[32],"v2"]