[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"amyloidosis-in-transthyretin-ttr\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:amyloidosis-in-transthyretin-ttr":24},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,43],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":17,"targetDuration":20,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":26,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100609339","comprehensive-program-for-hereditary-transthyretin-amyloidosis-100609339",false,"NCT07213297","Comprehensive Program for Hereditary Transthyretin Amyloidosis","Inclusion Criteria:\n\n-Participants with a pathogenic variant of the TTR gene (Hereditary Amyloidosis)\n\nExclusion Criteria:\n\n* wild-type TTR amyloidosis","ALL","18 Years",{"count":18,"type":19},20,"ESTIMATED","3 Years","OBSERVATIONAL","The Comprehensive Program for Hereditary Transthyretin Amyloidosis describes a prospective observational study focused on understanding hereditary transthyretin amyloidosis (ATTR), a progressive and potentially fatal condition marked by amyloid fibril deposits impacting multiple organs. The trial aims to characterize patient phenotypes, investigate factors affecting disease progression, and identify minimum criteria for disease onset. Conducted at Néstor Kirchner Hospital, the trial enrolls participants over 18 years old with confirmed pathogenic TTR variants. It includes thorough evaluations such as genetic testing sponsored by pharmaceutical companies, clinical assessments, and diverse diagnostic tests.",[24,25],"Amyloidosis in Transthyretin (TTR)","Amyloidosis, Familial",[27,28,29],"transthyretin","amyloidosis","comprensive care","RECRUITING","2026-03-20",{"date":33,"type":34},"2026-03-24","ACTUAL",{"date":36,"type":34},"2025-11-01",{"date":38,"type":19},"2028-12-01",{"name":40,"class":41},"Hospital de Alta Complejidad en Red","OTHER",1,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":52,"phases":53,"briefSummary":55,"conditions":56,"keywords":65,"overallStatus":72,"whyStopped":4,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":4},"100601897","phase-3-to-evaluate-the-long-term-safety-and-tolerability-of-acoramidis-in-participants-with-newly-diagnosed-attr-cm-act-early-ole-100601897","NCT07116473","To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)","A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)","Key Inclusion Criteria:\n\nParticipants must have completed the AG10-501 study (ACT-EARLY study) within the past 60 calendar days with a diagnosis of ATTR-CM (based on the AG10-501 protocol definition of ATTR-CM).\n\nKey Exclusion Criteria:\n\n1. Participants who completed the AG10-501 study with a diagnosis of ATTR-PN only, or who permanently discontinued study drug prior to diagnosis of ATTR-CM in AG10-501.\n2. History of AL or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).\n3. History of a monoclonal paraprotein or abnormal light chains in serum or urine (i.e., MGUS) in which AL has not been ruled out.\n4. Stage IV or V chronic kidney disease (corresponding to an eGFR ≤ 29 mL\u002Fmin\u002F1.73 m2), or undergoing renal dialysis, or recipient of a kidney transplant.\n5. Active malignancy, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated. In the event a participant developed a low-grade and treatable malignancy in the AG10-501 study (eg, low-grade, localized prostate cancer) and the decision by the Investigator at that time was to continue the participant in the AG10-501 study, that participant may still qualify for Study AG10-504 after documentation with the Medical Monitor.\n6. History of any organ transplant (with the exception of corneal transplant).\n7. Known hypersensitivity to acoramidis or any of the excipients within the study drug.\n8. Treatment for ATTR-CM with any ATTR-oriented on- or off-label or OTC product.",{"count":51,"type":19},250,"INTERVENTIONAL",[54],"PHASE3","The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).",[24,25,57,58,59,60,61,62,63,64],"Amyloid Cardiomyopathy","Amyloid Cardiomyopathy, Transthyretin-Related","Amyloidogenic Transthyretin (ATTR) Amyloidosis","Heart Disease","Cardiomyopathies","Amyloidosis, Hereditary, Transthyretin-Related","Polyneuropathies","Amyloidosis",[66,67,64,68,69,70,71],"ATTR-CM","ATTR-PN","Transthyretin","Amyloid","TTR","Hereditary","NOT_YET_RECRUITING","2025-08-07",{"date":75,"type":34},"2025-08-11",{"date":77,"type":19},"2025-11",{"date":79,"type":19},"2037-12",{"name":81,"class":82},"Eidos Therapeutics, a BridgeBio company","INDUSTRY"]