[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"transthyretin-amyloid-cardiomyopathy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:transthyretin-amyloid-cardiomyopathy":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,44,68,94,118,155],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100615856","phase-4-a-study-to-learn-more-about-the-change-in-the-blood-levels-of-transthyretin-when-participants-with-transthyretin-amyloidosis-with-cardiomyopathy-switch-from-tafamidis-to-acoramidis-100615856",false,"NCT07298044","A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis","A Prospective, Single-arm, Phase 4 Study to Evaluate the Course of Serum Transthyretin (TTR) Level With Acoramidis in Adult Patients With Variant or Wild-type Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) Previously Treated With Tafamidis","ACO-SWITCH","Inclusion Criteria:\n\n* Participants must be 18 to 90 years of age inclusive, at the time of signing the informed consent.\n* Diagnosis of ATTR-CM; disease defining examination, i.e., Single Photon Emission Computed Tomography (SPECT) or SPECT\u002FComputed Tomography (CT) or biopsy, within 24 months prior to Visit 1 (V1).\n* Participants must currently be treated with tafamidis and have used tafamidis for at least the previous 3 months prior to V1 and have adhered to tafamidis therapy.\n* New York Heart Association (NYHA) class ≤ II at V1.\n* Estimate glomerular filtration rate (eGFR) ≥ 30 mL\u002Fmin\u002F1.73m\\^2 at V1.\n* N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) \\> 300 and ≤ 7000 pg\u002FmL at V1.\n\nExclusion Criteria:\n\n* Prior liver or heart transplantation or planned within the next 12 months.\n* Current or planned use of ventricular assist device.\n* Active cancer or other disease that decreases the life expectancy to less than one year.\n* Heart failure due to ischemic heart disease.\n* Myocardial infarction, cardiovascular (CV) surgery, or unstable angina within the last 90 days prior to V1.\n* Confirmed diagnosis of light-chain amyloidosis.\n* Dialysis or severe renal impairment as reflected by Urinary Albumin Creatinine Ratio (UACR) \\> 300 mg\u002Fg at V1.\n* Major surgery 90 days prior to V1.\n* Recent initiation of Sodium-Glucose-Cotransporter-2 inhibitors (SGLT2i) within 3 months before V1.\n* Initiation of treatment with a diuretic or increase in diuretic dose within 3 months before V1.\n* Treatment with calcium channel blockers (e.g., verapamil, diltiazem) or digitalis.\n* Recent CV hospitalization within 3 months before V1.\n* Known hypersensitivity to acoramidis or to any of the excipients.\n* A condition that, as judged by the investigator, would preclude compliance with the study protocol, such as a history of substance abuse, alcoholism, or a psychiatric condition.\n* Known or suspected liver disorder and bile secretion\u002Fflow (cholestasis, also history of it).\n* Abnormal liver function tests at V1, defined as ALT (GPT) or AST (GOT) ≥ 3 x ULN or total bilirubin ≥ 3 x ULN at V1.","ALL","18 Years","90 Years",{"count":21,"type":22},50,"ESTIMATED","INTERVENTIONAL",[25],"PHASE4","Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid. TTR amyloid gets deposited in the heart wall and stops it from pumping blood properly, which may lead to heart failure. The breakage in TTR protein can be age-related (wild-type ATTR-CM), or genetic (variant ATTR-CM).\n\nThe study drug, acoramidis, works by attaching itself to the TTR protein, making TTR more stable and less likely to break down and form amyloid (clumps). This helps to slow down the progression of the disease, improve heart function, and increase the TTR levels in the blood. Acoramidis is an approved treatment for wild-type or variant ATTR-CM in Europe and the United States.\n\nTafamidis is another drug that stabilizes TTR and prevents it from breaking down.\n\nIn this study, acoramidis will be studied in participants with ATTR--CM who were previously treated with tafamidis. The main purpose of this study is to assess the change in blood TTR levels after participants are switched from tafamidis to acoramidis. This will be studied to understand if acoramidis causes an increase in blood TTR levels beyond the levels achieved with tafamidis.\n\nFor this, the researchers will measure the change in the levels of TTR protein in participants' blood after 6 months of the treatment with acoramidis, or earlier if a participant stops the treatment before reaching that six-month mark.\n\nAll participants will continue taking tafamidis during the screening period. In the treatment period of the study, participants will take acoramidis as two tablets twice daily by mouth, for up to 6 months.\n\nAt the start of this study, the study doctors will review each participant's medical history and check their overall health. The study doctors will perform electrocardiograms (ECG), and measure blood pressure and heart rate. Researchers will also take blood and urine samples from the participants to measure levels of TTr, NT-proBNP, hs-TnT, hs-CRP, RBP4, eGFR, creatinine, cystatin-C, UACR, and TSH at the start of the study, and at various time points thereafter (during the study) to assess heart, kidney and thyroid function.\n\nThere will be a total of 9 study check-ins. Participants will visit the study site twice: at screening and at the end of treatment period. A study nurse will visit the participant's home 6 times, at the start of treatment, Weeks 1, 2, 3 and 4, then again at 3 months. The final check-in will be done by phone.\n\nThe study doctors will monitor the health of the participants regularly for any medical problems during follow-up visits. Participants will know the treatment they will receive during the study. Each participant could be in the study for about 8 months.",[28],"Transthyretin Amyloid Cardiomyopathy",[30],"ATTR-CM","RECRUITING","2026-06-26",{"date":34,"type":35},"2026-06-29","ACTUAL",{"date":37,"type":35},"2026-02-13",{"date":39,"type":22},"2027-07-15",{"name":41,"class":42},"Bayer","INDUSTRY",20,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":17,"minAge":51,"maxAge":4,"enrollmentInfo":52,"targetDuration":4,"studyType":23,"phases":54,"briefSummary":56,"conditions":57,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":67},"100627437","phase-1-a-research-study-to-look-at-the-distribution-and-effects-of-coramitug-on-amyloid-deposits-in-heart-tissue-using-petct-imaging-in-people-with-attr-amyloidosis-100627437","NCT07448623","A Research Study to Look at the Distribution and Effects of Coramitug on Amyloid Deposits in Heart Tissue Using PET\u002FCT Imaging in People With ATTR Amyloidosis.","An Open-label Study to Evaluate the Biodistribution of 89Zr-coramitug and Investigate the Effects of Coramitug on Depleting TTR Amyloid Deposits in Myocardial Tissues Using PET\u002FCT Imaging in Participants With ATTR-CM","Inclusion Criteria:\n\n* Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.\n* Male or female.\n* Age greater than or equal to (≥) 60 years or above at the time of signing the informed consent.\n* For participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM):\n* Have an established diagnosis of ATTR-CM, with either wild-type Transthyretin (TTR) or variant TTR genotype (ATTRwt) or (ATTRv), with cardiac amyloid infiltration, increased left ventricular (LV) wall thickness, \\& heart failure (HF):\n\n  a) Cardiac amyloid infiltration demonstrated by: i. Cardiac biopsy positive for TTR amyloid, OR ii. Grade 2 or Grade 3 cardiac uptake at pyrophosphate\u002F3,3-diphosphono-1,2-propanodicarboxylic acid\u002Fhydroxymethylene diphosphonate (PYP\u002FDPD\u002FHMDP) scintigraphy with Single-Photon Emission Computed Tomography (SPECT\u002FCT) combined with an extracardiac biopsy positive for TTR amyloid, OR iii. Grade 2 or Grade 3 cardiac uptake at PYP\u002FDPD\u002FHMDP scintigraphy with SPECT\u002FCT combined with normal serum free light chain ratio \\& negative serum \\& urine immunofixation (Serum Immunofixation \\[SPIE\\] and Urine Immunofixation \\[UPIE\\]) Note: Bone tracer scintigraphy using 99m technetium (Tc)-labelled pyrophosphate (99mTc-PYP)\u002F99mTc-labelled 3,3-diphosphono-1,2- propanodicarboxylic acid (99mTc-DPD)\u002F99mTc-labeled hydroxymethylene diphosphonate (99m-Tc-HMDP) b. Increased LV wall thickness, as assessed by echocardiography showing LV posterior and septal wall thickness greater than or equal to (≥)13 millimeter (mm) for women and ≥ 14 mm for men (Note: Pre-existing echocardiogram up to 2 years old can be used).\n\n  c. Chronic HF with: i. At least 1 documented hospitalisation for HF occurring greater than (\\>) 3 months but less than (\\\u003C) 2 years, OR ii. History of HF manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath, signs of pulmonary con-gestion on x-ray or auscultation, or peripheral oedema) requiring ongoing treatment with a loop diuretic.\n\nExclusion Criteria:\n\n* Known or suspected hypersensitivity to study intervention(s) or related products.\n* Previous participation in this study. Participation is defined as signed informed consent.\n* Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential.\n* Current participation (i.e., signed informed consent) in any other interventional clinical study.\n* Participation in research studies involving exposure to radiation within a year preceding the screening period in this study.","60 Years",{"count":53,"type":22},14,[55],"PHASE1","The study is conducted in participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM), a heart disease that occurs in people with the disease ATTR amyloidosis. The purpose of this study is to see how radioactively labelled coramitug is taken up by the heart after administration through an infusion (Cohort 1), and to understand the extent to which coramitug can be displaced by radioactively labelled coramitug (Cohort 2). In this study it will also be investigated how safe coramitug is and how well it is tolerated when it is used by participants with ATTR-CM. Coramitug is potentially a new medicine for participants with ATTR-CM. Coramitug is a monoclonal antibody that potentially binds to the accumulations of the transthyretin protein and promotes its removal from the heart. It may also prevent the formation of clumps and may help with clearing existing clumps of the abnormal protein. The study will take a maximum of 85 days (for Cohort 1) or 106 days (for Cohort 2) when participating in Period A from the screening until the follow-up visit.",[28],"2026-04-07",{"date":60,"type":35},"2026-04-13",{"date":62,"type":35},"2026-03-02",{"date":64,"type":22},"2028-07-25",{"name":66,"class":42},"Novo Nordisk A\u002FS",1,{"id":69,"slug":70,"hasResults":11,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":4,"eligibilityCriteria":74,"healthyVolunteers":11,"sex":17,"minAge":75,"maxAge":76,"enrollmentInfo":77,"targetDuration":4,"studyType":79,"phases":4,"briefSummary":80,"conditions":81,"keywords":4,"overallStatus":83,"whyStopped":4,"lastUpdateSubmitDate":84,"lastUpdatePostDateStruct":85,"startDateStruct":87,"completionDateStruct":89,"leadSponsor":91,"locationsCount":4},"100606035","cardiac-amyloidosis-in-patients-with-aortic-stenosis-100606035","NCT07170306","Cardiac Amyloidosis in Patients With Aortic Stenosis","A Study on the Prevalence and Clinical Characteristics of Cardiac Amyloidosis in Patients With Aortic Stenosis","Inclusion Criteria:\n\n* Male or female patients aged ≥ 65 years and ≤ 85 years;\n* Patients with moderate-to-severe degenerative aortic stenosis (AS) who are hospitalized and scheduled to undergo transcatheter aortic valve replacement (TAVR);\n* Patients who voluntarily sign the Informed Consent Form (ICF) and are able to comply with the study-specified treatment plan, follow-up visits, laboratory tests, and other requirements.\n\nExclusion Criteria:\n\n* Aortic stenosis (AS) caused by congenital diseases or rheumatic immune diseases;\n* Severe renal impairment, chronic dialysis, or unresolved acute kidney injury after transcatheter aortic valve replacement (TAVR);\n* Terminal-stage diseases with an expected life span of \\\u003C 6 months;\n* Participation in other ongoing investigational studies of drugs or medical devices that have not yet been completed;\n* Patients who are unable to attend the follow-up visits scheduled in the study;\n* Failure to undergo TAVR surgery, transfer to surgical treatment, or in-hospital death.","65 Years","85 Years",{"count":78,"type":22},143,"OBSERVATIONAL","This study intends to conduct a prospective observation to investigate the prevalence of cardiac amyloidosis (CA) in patients with aortic stenosis (AS), compare the clinical characteristics between patients with isolated AS and those with AS complicated by CA (CA-AS), and simultaneously explore the impact of transcatheter aortic valve replacement (TAVR) on serum transthyretin (TTR) levels in patients with AS complicated by transthyretin amyloidosis (ATTR-AS), as well as its influence on the treatment outcomes of patients with isolated AS and ATTR-AS.",[82,28],"Aortic Stenosis","NOT_YET_RECRUITING","2026-01-15",{"date":86,"type":35},"2026-01-20",{"date":88,"type":22},"2026-01-31",{"date":90,"type":22},"2028-05-31",{"name":92,"class":93},"Shanghai Zhongshan Hospital","OTHER",{"id":95,"slug":96,"hasResults":11,"nctId":97,"briefTitle":98,"officialTitle":99,"acronym":4,"eligibilityCriteria":100,"healthyVolunteers":101,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":102,"targetDuration":4,"studyType":79,"phases":4,"briefSummary":104,"conditions":105,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":67},"100538512","phenotyping-and-characterization-of-wtattr-cm-trace-1-100538512","NCT06291805","Phenotyping and Characterization of wtATTR-CM (TRACE 1)","Phenotyping and Characterization of Danish Wild-type Transthyretin Amyloidosis Cardiomyopathy Patients: A Cross-sectional Study","Group 1: wtATTR-CM patients\n\nInclusion Criteria:\n\n* Patients \\> 18 years diagnosed with ATTRwt-CM by:\n* endomyocardial biopsy\n* DPD scintigraphy with Perugini grade 2-3 where variant amyloidosis is ruled out due to genetic testing.\n* Informed oral and written consent\n\nExclusion Criteria:\n\n* AL amyloidosis (light-chain amyloidosis).\n* Myelomatosis\n* Waldenström macroglobulinemia\n\nGroup 2: Control group\n\nInclusion Criteria:\n\n* Patients \\> 18 years\n* Informed oral and written consent\n\nExclusion Criteria:\n\n* Known cardiovascular disease including ischemic heart disease, heart failure, atrial fibrillation, presence of a pacemaker, or malignant hypertension. Well-controlled hypertension is acceptable.\n* Suspicion of cardiac amyloidosis assessed through clinical history, physical examination, ECG, and echocardiography focusing on \"red flags\":\n* Echocardiography with:\n* Myocardial hypertrophy (septum \\>11 mm)\n* Apical sparing in LV-GLS\n* Infiltrative changes in the right ventricle free wall, thickened atrioventricular valves, or thickened atrial septum\n* Symptoms of polyneuropathy\n* Low voltage on ECG or discrepancy between left ventricular thickness and ECG amplitude indicative of low voltage\n* Atrioventricular block (AV block)\n* Bilateral carpal tunnel syndrome\n* Surgery for spinal stenosis\n* Elevated troponin I or NT-pro-BNP",true,{"count":103,"type":22},120,"Descriptive cross-sectional study on 100 consecutive ATTRwt-CM patients reflecting all NAC stages aiming primarily to investigate ATTRwt-CM patient's quality of life (QoL) measures and their relation to ATTRwt-CM severity. Secondarily aiming to investigate the possibility to measure misTTR and fragTTR in plasma and urine and to detect fragTTR in endomyocardial biopsies from ATTRwt-CM patients. To investigate whether misTTR and fragTTR levels are correlated with ATTRwt-CM severity.",[106,28,107,108],"Transthyretin Amyloidosis","Wild-Type Transthyretin-Related (ATTR)Amyloidosis","Quality of Life","2025-08-12",{"date":111,"type":35},"2025-08-15",{"date":113,"type":35},"2024-02-20",{"date":115,"type":22},"2025-12",{"name":117,"class":93},"Steen Hvitfeldt Poulsen",{"id":119,"slug":120,"hasResults":11,"nctId":121,"briefTitle":122,"officialTitle":122,"acronym":4,"eligibilityCriteria":123,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":124,"targetDuration":4,"studyType":79,"phases":4,"briefSummary":126,"conditions":127,"keywords":144,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":146,"lastUpdatePostDateStruct":147,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":67},"100557519","flower-following-longitudinal-outcomes-with-epidemiology-for-rare-diseases-100557519","NCT06539169","FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","Inclusion Criteria:\n\n* Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:\n\nAlpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)\n\n\\- Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws\n\nExclusion Criteria:\n\n* Patient or LAR is unable to provide informed consent.\n* Patient resides in a country other than the United States and is unable to provide access to medical records.",{"count":125,"type":22},1000,"FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA\u002FRNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic\u002Fdigitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.",[128,129,130,131,132,133,134,135,136,137,138,139,140,141,142,28,143],"Alpha-Thalassemia","Beta-Thalassemia","Amyloidosis","Amyotrophic Lateral Sclerosis","Creutzfeld-Jakob Disease","Cystic Fibrosis","Duchenne Muscular Dystrophy","Early-Onset Alzheimer Disease","Ehlers-Danlos Syndrome","Huntington Disease","Gaucher Disease","GM1 Gangliosidosis","Myasthenia Gravis","Pompe Disease","Sickle Cell Disease","Rare Diseases",[145],"rare diseases","2024-11-12",{"date":148,"type":35},"2024-11-14",{"date":150,"type":35},"2024-06-10",{"date":152,"type":22},"2026-06-10",{"name":154,"class":42},"xCures",{"id":156,"slug":157,"hasResults":11,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":161,"eligibilityCriteria":162,"healthyVolunteers":11,"sex":17,"minAge":51,"maxAge":4,"enrollmentInfo":163,"targetDuration":4,"studyType":79,"phases":4,"briefSummary":165,"conditions":166,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":167,"lastUpdatePostDateStruct":168,"startDateStruct":170,"completionDateStruct":172,"leadSponsor":174,"locationsCount":67},"100523853","early-diagnosis-of-age-linked-cardiac-transthyretin-amyloidosis-by-selective-screening-in-spinal-stenosis-surgery-100523853","NCT06101108","Early Diagnosis of Age-Linked CArdiac TransThyRetin Amyloidosis by Selective Screening in Spinal Stenosis Surgery","Early Diagnosis of Age-Linked CArdiac TransThyRetin Amyloidosis by Selective Screening in Spinal Stenosis Surgery: the Multi-centric ALCATTRASS-study.","ALCATTRASS","Inclusion Criteria:\n\n* ≥ 60 years old\n* Lumbar spinal canal stenosis, undergoing spinal surgery\n* Westermark grade 3 or 4 ATTR amyloid burden on ligamentum flavum biopsy\n\nExclusion Criteria:\n\n* Prior diagnosis of cardiac amyloidosis\n* Unwilling to provide informed consent",{"count":164,"type":22},400,"The goal of this observational study is to keep track of the cardiological follow-up of patients who have undergone back surgery because of symptomatic narrowing of the spinal canal and in whom microscopic examination of the tissue removed during this surgery showed a high amount of local deposits of small proteins. This finding may be an early sign of a condition in which other organs (especially the heart) may also be affected by these proteins, called amyloidosis.\n\nPatients who participate in this study, will be followed up for life. During the first routine consultation with the cardiologist, the physician-investigator will collect information, such as demographics (age, weight, height, gender), medical history, medication, symptoms and the results of prescribed examinations. These examinations include microscopic examination of tissue removed during the surgery, blood tests, electrocardiography, echocardiography and a bone scan. These examinations are clinically necessary and appropriate, and patients should undergo them even without participating in the study.",[28],"2024-08-06",{"date":169,"type":35},"2024-08-07",{"date":171,"type":35},"2024-06-24",{"date":173,"type":22},"2026-08-01",{"name":175,"class":93},"Philippe Debonnaire"]