[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"metabolism-inborn-errors\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:metabolism-inborn-errors":45},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,77],{"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":84,"sex":16,"minAge":17,"maxAge":85,"enrollmentInfo":86,"targetDuration":4,"studyType":22,"phases":88,"briefSummary":90,"conditions":91,"keywords":4,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":5},"100542053","effect-of-large-neutral-amino-acids-in-adults-with-classical-phenylketonuria-100542053","NCT06337864","Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria","Safety and Efficacy of Treatment With Large Neutral Amino Acids in Patients With Classical Phenylketonuria","Patients ≥ 18 years of age with Classical PKU molecularly confirmed via the finding of two pathogenic variants in the phenylalanine hydroxylase (PAH) gene and\u002For historical evidence of Phe concentrations ≥1200 μmol\u002FL in the medical history\n\nInclusion Criteria:\n\n* Treatment initiation within the first month of life\n* Intelligence quotient over 84, based upon the baseline neuropsychological evaluation\n* Conventional dietary treatment up to minimum 15 years of age\n* Signed informed consent\n* Willing and able to comply with the protocol and study procedures\n\nExclusion Criteria:\n\n* Unable or unwilling to adhere to the requirements of the study\n* A female who is pregnant or breastfeeding or planning to get pregnant during the study period\n* Concomitant medication that may interfere with the PET analysis, as judged by the investigator\n* A serious neuropsychiatric disease that could interfere with the subject's ability to participate in the study at the discretion of the investigator\n* Concomitant treatment with BH4 supplementation (sapropterin) or Pegvaliase-pqpz (PALYNZIQ)\n* Failing to submit at least one blood Phe home sample during the year before study initiation\n* Standard MRI contraindications\n* Body weight over 110 kg",true,"50 Years",{"count":87,"type":21},30,[89],"NA","The overall aim of this study is to evaluate LNAA treatment as a potential alternative to conventional dietary treatment for PKU. This study investigates the effects of LNAA treatment compared to the classic dietary treatment on cerebral dopamine synthesis in patients with classic PKU. We will assess LNAAs effectiveness on neurotransmitter synthesis, cognitive function, mental health, and safety, compared to the standard diet.",[92,93,94,95,45,96,97,98],"Brain Diseases","Brain Diseases, Metabolic","Brain Diseases, Metabolic, Inborn","Genetic Diseases, Inborn","Amino Acid Metabolism, Inborn Errors","Metabolic Disease","Phenylketonurias","2025-04-02",{"date":101,"type":68},"2025-04-06",{"date":103,"type":68},"2024-11-01",{"date":105,"type":21},"2026-09-30",{"name":107,"class":108},"Rigshospitalet, Denmark","OTHER"]