33 completed, 18 recruiting, 9 unknown, 6 active not recruiting. 14 Phase 3, 8 Not applicable, 5 Phase 2, 4 Early Phase 1, 4 Phase 1.
Follow Amyloidosis, Hereditary, Transthyretin-Related: new trials by email Literature Review of Amyloidosis, Hereditary, Transthyretin-Related trials Research Report on Amyloidosis, Hereditary, Transthyretin-Related
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) Acoramidis (AG10) - Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial | Phase 3 | Not yet recruiting | Eidos Therapeutics, a BridgeBio company | 250 | 2026-11-01 | 2026-08-11 |
| Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations None ( observational study ) - Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy | Recruiting | AstraZeneca | 320 | 2026-09-01 | 2026-09-14 | |
| Retrospective Identification of Scintigraphic Cardiac Amyloidosis (RISCA) Cardiac Amyloidosis, Transthyretin Amyloidosis | Not yet recruiting | University Hospital, Lille | 57 | 2026-09-01 | 2026-05-15 | |
| Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis Acoramidis (AG10) oral tablet - Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis | Phase 4 | Recruiting | Eidos Therapeutics, a BridgeBio company | 150 | 2026-07-01 | 2026-07-29 |
| Concordance for Transthyretin Amyloidosis Between Synovial Biopsy and Anterior Carpal Ligament Biopsy Annular ligament biopsy - Carpal Tunnel, Transthyretin Amyloidosis | Not applicable | Recruiting | Clinique Saint Jean, France | 500 | 2026-05-27 | 2026-06-12 |
| Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis | Recruiting | University of Messina | 70 | 2026-01-30 | 2026-08-14 | |
| TEGSEDI Pregnancy Surveillance Program inotersen - Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy | Not yet recruiting | Akcea Therapeutics | 20 | 2025-12-01 | 2025-08-19 | |
| ATTRACT-52: Primary Care Cardiac Amyloidosis Screening in Ordu, Turkey Cardiac Amyloidosis, Transthyretin Amyloidosis | Enrolling by invitation | Kotyora Family Medicine Health Management and Education Association | 800 | 2025-12-01 | 2026-02-25 | |
| The Eplontersen Pregnancy and Lactation Outcomes Study Transthyretin Amyloidosis | Recruiting | AstraZeneca | 10 | 2025-11-30 | 2026-08-27 | |
| A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting Acoramidis (356 mg film-coated tablets) - Transthyretin Amyloid Cardiomyopathy (ATTR-CM) | Recruiting | Bayer | 2000 | 2025-10-29 | 2026-10-08 | |
| Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis. Amyloidosis in Transthyretin (TTR), Hereditary Amyloidosis, Transthyretin-Related | Withdrawn | Hospital General de Niños Pedro de Elizalde | 2025-06-20 | 2026-09-17 | ||
| Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant Acoramidis, Placebo oral tablet - Amyloidosis, Amyloid Cardiomyopathy | Phase 3 | Recruiting | Eidos Therapeutics, a BridgeBio company | 587 | 2025-05-12 | 2026-08-04 |
| A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Vutrisiran - Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Phase 3 | Enrolling by invitation | Alnylam Pharmaceuticals | 700 | 2024-12-03 | 2026-10-21 |
| A Phase 3 Study of NTLA-2001 in ATTRv-PN nexiguran ziclumeran, Normal Saline as Placebo - Neuromuscular Disease, Neuromuscular Diseases (NMD) | Phase 3 | Recruiting | Intellia Therapeutics | 60 | 2024-11-22 | 2026-04-16 |
| Exercise in Hereditary ATTR (ATTRv) Amyloidosis Exercise - Amyloidosis, Hereditary, Transthyretin-Related | Not applicable | Recruiting | University of Maia | 42 | 2024-10-01 | 2025-06-24 |
| Phenotypic Manifestations of Hereditary ATTR Amyloidosis A complete physical examination of all body systems, including height and body weight, Neurological examination includes motor strength testing; sensory testing with pinprick, light touch, temperature, and proprioception; deep tendon reflexes; and gait assessment., Electrocardiogram (12-lead ECG) - Hereditary Amyloidosis, Transthyretin-Related | Recruiting | Hospital 9 de Julio de Las Breñas | 57 | 2024-09-01 | 2026-04-02 | |
| Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis Treatment of transthyretin (ATTR) amyloidosis in observational study setting - Transthyretin Amyloidosis, ATTR-CM | Recruiting | AstraZeneca | 1850 | 2024-06-25 | 2026-09-23 | |
| An Interventional Pilot Study to Investigate the Feasibility and Acceptance of a Structured Psychological Support Program for Patients, Caregivers, and Presymptomatic Carriers in Hereditary Transthyretin Amyloidosis With Cardiomyopathy Psychological Support - Hereditary Transthyretin Amyloidosis | Not applicable | Recruiting | Fondazione IRCCS Policlinico San Matteo di Pavia | 150 | 2024-05-24 | 2025-04-06 |
| Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy Standard of Care - Hereditary Amyloidosis, Transthyretin-Related, Asymptomatic Carrier State | Active not recruiting | Alnylam Pharmaceuticals | 346 | 2024-04-25 | 2026-10-15 | |
| Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran Six minutes walk test, Kansas City questionnaire, COMPASS31 self questionnaire - Transthyretin Amyloidosis, Amyloidosis, Hereditary | Active not recruiting | Rennes University Hospital | 20 | 2024-04-12 | 2026-01-05 |
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Amyloidosis (34) · Amyloid Neuropathies, Familial (29) · Amyloidosis, Familial (18) · Cardiomyopathies (18) · Polyneuropathies (14) · Alzheimer Disease (10) · Amyloid Neuropathies (10) · Carpal Tunnel Syndrome (4) · Heart Failure (4) · Immunoglobulin Light-chain Amyloidosis (4) · Aortic Valve Stenosis (3) · Heart Diseases (3) · Neuromuscular Diseases (3) · Peripheral Nervous System Diseases (3)
Nutritional and Metabolic Diseases (53) · Metabolic Diseases (51) · Proteostasis Deficiencies (51) · Nervous System Diseases (36) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (34) · Neuromuscular Diseases (34) · Peripheral Nervous System Diseases (33) · Genetic Diseases, Inborn (32) · Metabolism, Inborn Errors (32) · Neurodegenerative Diseases (28) · Heredodegenerative Disorders, Nervous System (27) · Cardiovascular Diseases (22) · Amyloid Neuropathies (20) · Heart Diseases (19) · Amyloidosis (18)
patisiran (7) · attruby (6) · tafamidis (4) · Doxycycline (3) · Biopsy (2) · Blood Specimen Collection (2) · Inotersen (2) · NTLA-2001 (2) · ursodoxicoltaurine (2) · Amyloid (1) · Data Collection (1) · Echocardiography (1) · Echocardiography, Doppler, Color (1) · Electrocardiography (1) · Electrocardiography, Ambulatory (1)
Alnylam Pharmaceuticals (12) · Eidos Therapeutics, a BridgeBio company (6) · AstraZeneca (5) · The Cleveland Clinic (4) · Akcea Therapeutics (3) · Pfizer (3) · CENTOGENE GmbH Rostock (2) · Fondazione IRCCS Policlinico San Matteo di Pavia (2) · Intellia Therapeutics (2) · Steen Hvitfeldt Poulsen (2) · University of Messina (2) · University of Texas Southwestern Medical Center (2) · Yale University (2) · Assistance Publique Hopitaux De Marseille (1) · Attralus, Inc. (1)
United States (36) · France (19) · Italy (18) · Germany (17) · Spain (16) · Portugal (15) · United Kingdom (15) · Sweden (12) · Argentina (11) · Brazil (11) · Canada (11) · Denmark (11) · Belgium (10) · Netherlands (10) · South Korea (9)
Mazen A Hanna, MD (3) · Mark McGovern, RN, CCRN (2) · Arndt Rolfs, Prof. (1) · Brian Drachman (1) · Chafic Karam, MD (1) · Edward J Miller, MD (1) · Edward Miller, MD (1) · Elisabete Martins, MD, PhD (1) · Erwan Donal (1) · Giampaolo Merlini, MD (1) · Gianluca Di Bella, MD, PhD (1)
PMID 30938420, PMID 39213194, PMID 11488533, PMID 16631014, PMID 18382889, PMID 18579156, PMID 22056963, PMID 23193944, PMID 2394453, PMID 25091367, PMID 25372741, PMID 25551524, PMID 26209803, PMID 26224076, PMID 26870387, PMID 27386769, PMID 27652282, PMID 28384285, PMID 28893208, PMID 29019612, PMID 29048471, PMID 29305186, PMID 29633228, PMID 29972753, PMID 30010028, PMID 30095083, PMID 30145929, PMID 30336828, PMID 30343093, PMID 30586695, PMID 30810593, PMID 30878017, PMID 30915522, PMID 31140154, PMID 31163298, PMID 31215818, PMID 31322739, PMID 31353964, PMID 31400950, PMID 31480867, PMID 31726319, PMID 32062791, PMID 32078382, PMID 32169556, PMID 32400202, PMID 32430600, PMID 32936265, PMID 33025472, PMID 33725199, PMID 33982288
2018 (4), 2023 (3), 2015 (1), 2019 (1), R01HL160892 (1)