{"id":"treatment-for-ttr-amyloidosis","rwe":[],"tags":[],"phase":"preclinical","safety":{"boxedWarnings":[],"safetySignals":[],"drugInteractions":[],"commonSideEffects":[],"contraindications":[],"specialPopulations":{},"discontinuationRates":[],"seriousAdverseEvents":[]},"status":"active","trials":["NCT05560555","NCT06651073","NCT06338696","NCT00935012","NCT00628745","NCT01604122","NCT03190577","NCT00409175","NCT04828993","NCT00630864","NCT02791230","NCT00925002","NCT06321523","NCT04456582","NCT03842163","NCT00694161","NCT05374564","NCT04108091","NCT02146378","NCT01435655","NCT04814186","NCT06086353","NCT04801329","NCT00791492","NCT04535349"],"aliases":[],"patents":[],"pricing":[],"allNames":"treatment for ttr amyloidosis","offLabel":[],"timeline":[{"date":"2008","type":"neutral","milestone":"Phase 3 Initiated","description":"Phase 3 trial (An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patien) — Familial Amyloid Polyneuropathy"},{"date":"2010","type":"neutral","milestone":"Phase 2 Initiated","description":"Phase 2 trial (The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients Wit) — Transthyretin-associated Amyloidosis With Polyneuropathy"},{"date":"2023","type":"neutral","milestone":"Phase 4 Initiated","description":"Phase 4 trial (The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients) — Transthyretin Amyloid Polyneuropathy (ATTR-PN)"},{"date":"2024","type":"neutral","milestone":"Phase 1 Initiated","description":"Phase 1 trial (Parametric Cardiac 18F-flutemetamol PET Imaging in ATTR Cardiomyopathy) — Cardiomyopathies, Primary"}],"aiSummary":"Treatment for TTR amyloidosis includes the use of small molecule interventions such as Tafamidis and ALN-TTRSC (revusiran), which are administered subcutaneously. Cardiac Contractility Modulation (CCM) therapy is also being studied as a potential treatment option for patients with TTR amyloidosis and heart failure.","brandName":"Treatment for TTR amyloidosis","companyId":"pfizer","ecosystem":[],"mechanism":{"target":"","novelty":"","modality":"","drugClass":"The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if n","explanation":"","oneSentence":"The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if n","technicalDetail":""},"commercial":null,"references":[],"biosimilars":[],"companyName":"Pfizer Inc.","competitors":[],"genericName":"treatment-for-ttr-amyloidosis","indications":{"approved":[],"offLabel":[],"pipeline":[{"name":"Transthyretin (TTR) Amyloid Cardiomyopathy","phase":"preclinical","trialId":"","patients":null,"diseaseId":"cardiovascular","trialName":"","primaryEndpoint":"","expectedCompletion":""}]},"labelChanges":[],"relatedDrugs":[],"trialDetails":[{"nctId":"NCT05560555","phase":"N/A","title":"Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":5,"indication":"Hereditary Transthyretin Amyloidosis (ATTRv)","completionDate":"2022-11","primaryEndpoint":"A study of patients with hereditary transthyretin amyloidosis (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt) that have been enrolled in B3461028 and B3461045 studies in Spain - exposed to ta"},{"nctId":"NCT06651073","phase":"N/A","title":"ATTR-CM: A Multi-country, Non-interventional Disease Registry","status":"Active Not Recruiting","sponsor":"Pfizer","isPivotal":false,"enrollment":350,"indication":"Transthyretin Amyloid Cardiopathy","completionDate":"2028-04","primaryEndpoint":"This study is a multi-center, non-interventional, disease registry to characterize the natural history of ATTR-CM and treatment patterns in clinically diagnosed ATTR-CM patients."},{"nctId":"NCT06338696","phase":"N/A","title":"Thailand ATTR-CM Registry","status":"Recruiting","sponsor":"Mahidol University","isPivotal":false,"enrollment":105,"indication":"Transthyretin Amyloid Cardiomyopathy (ATTR-CM)","completionDate":"2029-06","primaryEndpoint":"The investigators of this registry aim to study the natural history, clinical presentation, characteristics, and imaging findings of patients diagnosed with ATTR amyloidosis in Thailand longitudinally"},{"nctId":"NCT00935012","phase":"Phase 3","title":"Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) A","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":31,"indication":"ATTR-CM","completionDate":"2019-11","primaryEndpoint":"Open-label Safety and Efficacy Evaluation of Fx-1006a in Patients with V122i Or Wild-type Transthyretin (ttr) Amyloid Cardiomyopathy."},{"nctId":"NCT00628745","phase":"N/A","title":"Transthyretin Amyloidosis Outcome Survey (THAOS)","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":6718,"indication":"Transthyretin Gene Mutations","completionDate":"2023-06","primaryEndpoint":"THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-typ"},{"nctId":"NCT01604122","phase":"N/A","title":"Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":1,"indication":"Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)","completionDate":"2015-07","primaryEndpoint":"This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers."},{"nctId":"NCT03190577","phase":"N/A","title":"Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy o","status":"Completed","sponsor":"Nantes University Hospital","isPivotal":false,"enrollment":400,"indication":"Familial Amyloid Neuropathy","completionDate":"2022-05","primaryEndpoint":"Familial amyloid neuropathy due to transthyretin gene mutations (TTR-FAP) is a rare autosomal dominant inherited disease resulting in the abnormal multi-system deposition of amyloid proteins."},{"nctId":"NCT00409175","phase":"Phase 2/3","title":"Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":128,"indication":"Familial Amyloid Polyneuropathy","completionDate":"2009-05","primaryEndpoint":"This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy (FAP)."},{"nctId":"NCT04828993","phase":"Phase 4","title":"The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":15,"indication":"Transthyretin Amyloid Polyneuropathy (ATTR-PN)","completionDate":"2023-02","primaryEndpoint":"This is a single-arm, open-label, multicenter study designed to evaluate the efficacy, safety, tolerability as well as pharmacodynamics of tafamidis meglumine in ATTR-PN participants in China."},{"nctId":"NCT00630864","phase":"Phase 2","title":"The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients Wit","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":21,"indication":"Transthyretin-associated Amyloidosis With Polyneuropathy","completionDate":"2010-01","primaryEndpoint":"This is an open-label, multicenter, international study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients with non-V"},{"nctId":"NCT02791230","phase":"Phase 3","title":"Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":1733,"indication":"Transthyretin (TTR) Amyloid Cardiomyopathy","completionDate":"2023-10","primaryEndpoint":"Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy"},{"nctId":"NCT00925002","phase":"Phase 3","title":"Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":93,"indication":"ATTR-PN","completionDate":"2020-07","primaryEndpoint":"This is a Phase 3, open-label study designed to obtain additional long-term safety and efficacy data for oral tafamidis (20 mg soft gelatin capsule) administered once daily (QD)."},{"nctId":"NCT06321523","phase":"N/A","title":"A Study to Learn About the Study Medicine Called Tafamidis 61mg in People Diagnosed With Transthyret","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":13,"indication":"Transthyretin Amyloid Cardiomyopathy","completionDate":"2025-10","primaryEndpoint":"The purpose of this clinical trial is to learn about the effects of the study medicine (called Tafamidis 61milligrams (mg)) for the potential treatment of Transthyretin amyloid cardiomyopathy (ATTR-CM"},{"nctId":"NCT04456582","phase":"N/A","title":"Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Bidimensional Shear W","status":"Unknown","sponsor":"Fábio Fernandes","isPivotal":false,"enrollment":60,"indication":"Amyloidosis","completionDate":"2021-01","primaryEndpoint":"Amyloidosis by mutation of the transthyretin gene (ATTRh) is part of a group of diseases in which the deposit of structurally abnormal proteins (amyloid fibrils) affects multiple organs such as: liver"},{"nctId":"NCT03842163","phase":"N/A","title":"Prevalence and Characteristics of Transthyretin Amyloidosis in Patients With Left Ventricular Hypert","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":812,"indication":"Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)","completionDate":"2022-06","primaryEndpoint":"The main purpose of this study is to determine the prevalence of ATTR Cardiomyopathy among patients admitted due to Left Ventricular Hypertrophy (LVH) \\>15mm of unknown etiology by using a 99mTc-trace"},{"nctId":"NCT00694161","phase":"Phase 2","title":"The Effects Of Fx-1006A On Transthyretin Stabilization And Clinical Outcome Measures In Patients Wit","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":35,"indication":"Cardiomyopathy","completionDate":"2010-01","primaryEndpoint":"Open-label, multicenter, international, single-treatment study designed to determine TTR stabilization as well as Fx-1006A safety and tolerability, and its effects on clinical outcomes in patients wit"},{"nctId":"NCT05374564","phase":"Phase 1","title":"Parametric Cardiac 18F-flutemetamol PET Imaging in ATTR Cardiomyopathy","status":"Completed","sponsor":"Yale University","isPivotal":false,"enrollment":12,"indication":"Cardiomyopathies, Primary","completionDate":"2024-11","primaryEndpoint":"18F-Flutemetamol (Vizamyl) is a radioactive diagnostic agent indicated and FDA-approved for Positron Emission Tomography (PET) imaging of the brain to estimate β-amyloid neuritic plaque density in adu"},{"nctId":"NCT04108091","phase":"N/A","title":"Vyndaqel Capsules Special Investigation (ATTR-CM)","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":1214,"indication":"Transthyretin (TTR) Amyloid Cardiomyopathy","completionDate":"2025-03","primaryEndpoint":"Secondary Data Collection : To confirm the safety and effectiveness profiles under the actual medical practice of Vyndaqel in Japan."},{"nctId":"NCT02146378","phase":"N/A","title":"Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":500,"indication":"Transthyretin Familial Amyloid Polyneuropathy","completionDate":"2023-03","primaryEndpoint":"The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions \\[ADRs\\]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel"},{"nctId":"NCT01435655","phase":"Phase 3","title":"The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M ","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":10,"indication":"Transthyretin Familial Amyloid Polyneuropathy","completionDate":"2014-02","primaryEndpoint":"Tafamidis has been developed as an oral specific stabilizer of transthyretin tetramer."},{"nctId":"NCT04814186","phase":"Phase 4","title":"A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Am","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":53,"indication":"Transthyretin Amyloid Cardiomyopathy","completionDate":"2023-10","primaryEndpoint":"This is a national, multi-center, single-arm study, open-label to patients with symptomatic Transthyretin amyloid cardiomyopathy (ATTR-CM) who are tafamidis naïve."},{"nctId":"NCT06086353","phase":"N/A","title":"A Study to Learn About Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in ","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":6,"indication":"Transthyretin Amyloid Cardiomyopathy","completionDate":"2025-06","primaryEndpoint":"The purpose of this study is to learn about the safety of Tafamidis for the treatment of Transthyretin amyloid cardiomyopathy (ATTR-CM) in India."},{"nctId":"NCT04801329","phase":"N/A","title":"Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid Car","status":"Active Not Recruiting","sponsor":"Pfizer","isPivotal":false,"enrollment":110,"indication":"ATTR-CM (Transthyretin Amyloid Cardiomyopathy)","completionDate":"2026-12","primaryEndpoint":"This non-interventioal study will be performed by design of post-marketing surveillance (PMS) as an additional pharmacovigilance activity of the Risk Management Plan (RMP) for Vyndamax® Capsules, whic"},{"nctId":"NCT00791492","phase":"Phase 2/3","title":"An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patien","status":"Completed","sponsor":"Pfizer","isPivotal":false,"enrollment":86,"indication":"Familial Amyloid Polyneuropathy","completionDate":"2008-08","primaryEndpoint":"This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN."},{"nctId":"NCT04535349","phase":"N/A","title":"Quantitative Analysis of Myocardial Uptake of Bone Radiopharmaceuticals in Patients With Cardiac ATT","status":"Recruiting","sponsor":"University Hospital, Caen","isPivotal":false,"enrollment":35,"indication":"Amyloidosis Transthyretin","completionDate":"2025-06","primaryEndpoint":"Introduction: Transthyretin cardiac amyloidosis (ATTR) is an important cause of heart failure."}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"administration":{"icon":"","route":"","frequency":"","formulation":""},"crossReferences":{"chemblId":"CHEMBL521875"},"formularyStatus":[],"developmentCodes":[],"ownershipHistory":[],"therapeuticAreas":["Cardiovascular"],"biosimilarFilings":[],"firstApprovalDate":"","companionDiagnostics":[],"firstApprovalCountry":null,"genericManufacturerList":[],"modality":"","enrichmentLevel":3,"visitCount":3,"trialStats":{"total":1,"withResults":0},"verificationStatus":"partial","dataCompleteness":{"mechanism":true,"indications":false,"safety":false,"trials":true,"score":2}}