[{"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:":134},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,77,110],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":47,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":76},"100567749","phase-3-a-phase-3-study-of-ntla-2001-in-attrv-pn-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":20,"type":21},60,"ESTIMATED","INTERVENTIONAL",[24],"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.",[27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46],"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",[48,39,49,50,51,52,53,54,55,56,57,58,59,60,61,62,63],"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":67,"type":68},"2026-04-16","ACTUAL",{"date":70,"type":68},"2024-11-22",{"date":72,"type":21},"2028-08",{"name":74,"class":75},"Intellia Therapeutics","INDUSTRY",14,{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":4,"eligibilityCriteria":83,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":84,"enrollmentInfo":85,"targetDuration":4,"studyType":22,"phases":87,"briefSummary":88,"conditions":89,"keywords":91,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":108,"locationsCount":109},"100525968","phase-3-magnitude-a-phase-3-study-of-ntla-2001-in-participants-with-transthyretin-amyloidosis-with-cardiomyopathy-attr-cm-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":86,"type":21},1200,[24],"To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.",[90],"Transthyretin Amyloidosis (ATTR) With Cardiomyopathy",[48,39,92,50,93,54,51,55,38,94,95,96,97,98,99,100],"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":103,"type":68},"2026-03-27",{"date":105,"type":68},"2023-12-13",{"date":107,"type":21},"2028-04",{"name":74,"class":75},132,{"id":111,"slug":112,"hasResults":11,"nctId":113,"briefTitle":114,"officialTitle":115,"acronym":4,"eligibilityCriteria":116,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":117,"targetDuration":4,"studyType":119,"phases":4,"briefSummary":120,"conditions":121,"keywords":4,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":125,"lastUpdatePostDateStruct":126,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":133},"100492877","long-term-follow-up-ltfu-of-subjects-dosed-with-ntla-2001-100492877","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":118,"type":21},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.",[122,123,124],"Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy","Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy","Wild-Type Transthyretin Cardiac Amyloidosis","2025-08-20",{"date":127,"type":68},"2025-08-26",{"date":129,"type":68},"2023-07-10",{"date":131,"type":21},"2038-03",{"name":74,"class":75},4,""]