[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Eidos Therapeutics, a BridgeBio company\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":81},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,52],{"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":33,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100559420","phase-3-acoramidis-transthyretin-amyloidosis-prevention-trial-in-the-young-act-early-study-in-asymptomatic-carriers-of-a-pathogenic-ttr-variant-100559420",false,"NCT06563895","Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant","A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)","Key Inclusion Criteria:\n\n* Male or female ≥ 18 to ≤ 75 years of age inclusive.\n* Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M\u002Fp.V50M, V122I\u002Fp.V142I, T60A\u002Fp.T80A, or all other pathogenic TTR variants).\n* Participant's age is within 10 years younger than or older than PADO.\n\nKey Exclusion Criteria:\n\n* Evidence of ATTR-CM or ATTR-PN.\n* Current or past (within last 1 to 12 months, depending on specific agent) treatment with other TTR modifying therapies.\n* Contraindication to or inability to undergo cardiac magnetic resonance testing.\n* Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy\n* Other diseases or conditions such has cancer within 5 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV.\n* Major surgery within the past 3 months or planned during the next 12 months.\n* Known hypersensitivity to acoramidis.","ALL","18 Years","75 Years",{"count":20,"type":21},587,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age.\n\nAmyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.\n\nAcoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.\n\nThis study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.",[27,28,29,30,31,32],"Amyloidosis","Amyloid Cardiomyopathy","Transthyretin Amyloidosis","Cardiomyopathies","Heart Diseases","Polyneuropathies",[27,34,35,36,37,38],"ATTR-CM","ATTR-PN","Transthyretin","Amyloid","TTR","RECRUITING","2026-06-25",{"date":42,"type":43},"2026-06-29","ACTUAL",{"date":45,"type":43},"2025-05-12",{"date":47,"type":21},"2032-12",{"name":49,"class":50},"Eidos Therapeutics, a BridgeBio company","INDUSTRY",103,{"id":53,"slug":54,"hasResults":11,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":4,"eligibilityCriteria":58,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":59,"targetDuration":4,"studyType":22,"phases":61,"briefSummary":62,"conditions":63,"keywords":70,"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":60,"type":21},250,[24],"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).",[64,65,28,66,67,68,30,69,32,27],"Amyloidosis in Transthyretin (TTR)","Amyloidosis, Familial","Amyloid Cardiomyopathy, Transthyretin-Related","Amyloidogenic Transthyretin (ATTR) Amyloidosis","Heart Disease","Amyloidosis, Hereditary, Transthyretin-Related",[34,35,27,36,37,38,71],"Hereditary","NOT_YET_RECRUITING","2025-08-07",{"date":75,"type":43},"2025-08-11",{"date":77,"type":21},"2025-11",{"date":79,"type":21},"2037-12",{"name":49,"class":50},""]