[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Intellia Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":131},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,76,108],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":46,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":75},"100567749",false,"NCT06672237","A Phase 3 Study of NTLA-2001 in ATTRv-PN","MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)","Inclusion Criteria:\n\n* Diagnosis of ATTRv-PN\n* Karnofsky Performance Status (KPS) ≥ 60\n\nExclusion Criteria:\n\n* Other causes of amyloidosis (amyloidosis caused by non-TTR protein)\n* Other known causes of sensorimotor or autonomic neuropathy\n* Diabetes mellitus\n* New York Heart Association Class III or IV heart failure\n* Liver failure\n* Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection\n* Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))\n* Estimated Glomerular Filtration Rate \\\u003C 30 mL\u002Fmin\u002F1.73 m2\n* Unable or unwilling to take vitamin A supplementation for the duration of the study\n* History of liver disease","ALL","18 Years","85 Years",{"count":19,"type":20},60,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.",[26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45],"Neuromuscular Disease","Neuromuscular Diseases (NMD)","Neurodegenerative Disease","Neurodegenerative Disease, Hereditary","Neurodegenerative Diseases","Neuromuscular Diseases","Nerve Disorders","Nervous System Disease","Nervous System Diseases","Genetic Disease, Inborn","Amyloidosis, Familial","Amyloidosis, Hereditary","Amyloidosis","Polyneuropathies","Amyloid Neuropathies","Amyloid Neuropathies, Familial","Peripheral Nervous System Disease","Peripheral Nervous System Diseases","Metabolism, Inborn Errors","Metabolic Diseases",[47,38,48,49,50,51,52,53,54,55,56,57,58,59,60,61,62],"TTR","Polyneuropathy","NTLA-2001","ATTR","ATTR-PN","ATTRv-PN","Transthyretin","TTR-mediated amyloidosis","Amyloidosis, hereditary","Amyloidosis, hereditary, transthyretin-related amyloidosis","Transthretin amyloid polyneuropathy","TTR PN","TTR polyneuropathy","nexiguran ziclumeran","nex-z","CRISPR","RECRUITING","2026-04-13",{"date":66,"type":67},"2026-04-16","ACTUAL",{"date":69,"type":67},"2024-11-22",{"date":71,"type":20},"2028-08",{"name":73,"class":74},"Intellia Therapeutics","INDUSTRY",14,{"id":77,"slug":4,"hasResults":10,"nctId":78,"briefTitle":79,"officialTitle":80,"acronym":4,"eligibilityCriteria":81,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":82,"enrollmentInfo":83,"targetDuration":4,"studyType":21,"phases":85,"briefSummary":86,"conditions":87,"keywords":89,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":107},"100525968","NCT06128629","MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With 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","90 Years",{"count":84,"type":20},1200,[23],"To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.",[88],"Transthyretin Amyloidosis (ATTR) With Cardiomyopathy",[47,38,90,49,91,53,50,54,37,92,93,94,95,96,97,98],"Cardiomyopathy","ATTR-CM","Amyloidosis, Hereditary, Transthyretin-Related Amyloidosis","Transthyretin amyloid cardiomyopathy","TTR cardiomyopathy","Wild-type TTR","V122I","Amyloidosis, Wild Type","CRISPR\u002FCas9","2026-03-23",{"date":101,"type":67},"2026-03-27",{"date":103,"type":67},"2023-12-13",{"date":105,"type":20},"2028-04",{"name":73,"class":74},132,{"id":109,"slug":4,"hasResults":10,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":4,"eligibilityCriteria":113,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":114,"targetDuration":4,"studyType":116,"phases":4,"briefSummary":117,"conditions":118,"keywords":4,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":130},"100492877","NCT05697861","Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001","Long-Term Follow-Up of Subjects Treated With NTLA-2001","Inclusion Criteria:\n\n1. A subject has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received.\n2. A subject has provided informed consent for the LTFU study.\n\nExclusion Criteria:\n\nNone",{"count":115,"type":20},72,"OBSERVATIONAL","This is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.",[119,120,121],"Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy","Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy","Wild-Type Transthyretin Cardiac Amyloidosis","2025-08-20",{"date":124,"type":67},"2025-08-26",{"date":126,"type":67},"2023-07-10",{"date":128,"type":20},"2038-03",{"name":73,"class":74},4,""]