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Cardiomyopathy (ATTR-CM)","MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)","Inclusion Criteria:\n\n* Documented diagnosis of ATTR amyloidosis with cardiomyopathy\n* Medical history of heart failure (HF)\n* Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention\n* Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg\u002FmL and less than 10,000 pg\u002FmL\n\nExclusion Criteria:\n\n* New York Heart Association (NYHA) Class IV HF\n* Polyneuropathy Disability score of IV (confined to wheelchair or bed)\n* Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection\n* History of active malignancy within 3 years prior to screening\n* RNA silencer therapy (patisiran, inotersen and\u002For eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed\n* Initiation of tafamidis or acoramidis within 56 days prior to study dosing\n* Estimated glomerular filtration rate (eGFR) \\\u003C30 mL\u002Fmin\u002F1.73m\\^2\n* History of liver disease\n* Uncontrolled blood pressure\n* Unable or unwilling to take vitamin A supplementation for the duration of the study","ALL","18 Years","90 Years",{"count":1190,"type":1191},1200,"ESTIMATED","INTERVENTIONAL",[1194],"PHASE3","To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.",[1197],"Transthyretin Amyloidosis (ATTR) With Cardiomyopathy",[1199,1200,1201,34,1202,1203,1204,1205,1206,1207,1208,1209,1210,1211,1212,1213],"TTR","Amyloidosis","Cardiomyopathy","ATTR-CM","Transthyretin","ATTR","TTR-mediated amyloidosis","Amyloidosis, Hereditary","Amyloidosis, Hereditary, Transthyretin-Related Amyloidosis","Transthyretin amyloid cardiomyopathy","TTR cardiomyopathy","Wild-type TTR","V122I","Amyloidosis, Wild Type","CRISPR\u002FCas9","2026-03-23",{"date":1216,"type":1217},"2026-03-27","ACTUAL",{"date":1219,"type":1217},"2023-12-13",{"date":1221,"type":1191},"2028-04",{"name":5,"class":6},132]