20 completed, 9 recruiting, 7 unknown, 2 withdrawn. 5 Not applicable, 4 Early Phase 1, 4 Phase 2, 4 Phase 3, 2 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 5 | Completed 3, Recruiting 2 |
| Early Phase 1 | 4 | Completed 3, Withdrawn 1 |
| Phase 2 | 4 | Completed 3, Recruiting 1 |
| Phase 3 | 4 | Completed 3, Recruiting 1 |
| Phase 4 | 2 | Recruiting 1, Unknown 1 |
Follow Transthyretin Amyloidosis: new trials by email Literature Review of Transthyretin Amyloidosis trials Research Report on Transthyretin Amyloidosis
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 | |
| 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 |
| 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 | |
| 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 | |
| Phenotyping and Characterization of wtATTR-CM (TRACE 1) Transthyretin Amyloidosis, Transthyretin Amyloid Cardiomyopathy | Recruiting | Steen Hvitfeldt Poulsen | 120 | 2024-02-20 | 2025-08-15 | |
| Prevalence of Transthyretin Cardiac Amyloidosis in Clinically Significant Aortic Stenosis 99mTc-PYP - Aortic Stenosis, Transthyretin Amyloidosis | Early Phase 1 | Completed | Mayo Clinic | 4 | 2023-07-14 | 2024-08-07 |
| New Biomarkers and Plasma Prothrombotic Potential in Cardiac Transthyretin Amyloidosis blood test - Transthyretin Amyloidosis | Unknown | John Paul II Hospital, Krakow | 100 | 2023-07-11 | 2024-04-19 | |
| Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis 124I-Evuzamitide - Amyloidosis, Transthyretin Amyloidosis | Phase 2 | Recruiting | Oregon Health and Science University | 150 | 2023-01-10 | 2026-04-13 |
| Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy patisiran - Polyneuropathies, Wild Type ATTR Amyloidosis | Early Phase 1 | Completed | Austin Neuromuscular Center | 10 | 2022-08-03 | 2026-05-22 |
| Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records Computer algorithm for ATTR - Transthyretin Amyloidosis | Early Phase 1 | Withdrawn | Yale University | 2022-08-01 | 2025-09-18 | |
| Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR I 124-Evuzamitide - Transthyretin Amyloidosis | Phase 2 | Completed | Columbia University | 10 | 2022-07-08 | 2025-07-18 |
| Study of Cerebral MRI Anomalies in Mutated Transthyretin Amyloidosis Patients No intervention, - Transthyretin Amyloidosis | Completed | Centre Hospitalier Universitaire de Nīmes | 36 | 2021-10-25 | 2022-09-02 | |
| The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients Tafamidis Pill - Transthyretin Amyloidosis | Phase 4 | Unknown | Peking University Third Hospital | 10 | 2021-06-01 | 2021-07-15 |
| Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF 99mTc-pyrophosphate Scintigraphy - Transthyretin Amyloidosis, Heart Failure | Not applicable | Recruiting | Massachusetts General Hospital | 515 | 2020-10-02 | 2026-09-01 |
| Transthyretin Cardiac Amyloidosis in Patients With Idiopathic Carpal Tunnel Syndrome Referred for Release Surgery Descriptive cohort - Transthyretin Amyloidosis, Carpal Tunnel Syndrome | Terminated | University Hospital, Toulouse | 6 | 2020-10-01 | 2024-02-20 |
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Amyloidosis, Hereditary, Transthyretin-Related (42) · Amyloidosis (15) · Amyloid Neuropathies, Familial (13) · Cardiomyopathies (8) · Alzheimer Disease (5) · Amyloidosis, Familial (4) · Polyneuropathies (4) · Amyloid Neuropathies (3) · Carpal Tunnel Syndrome (3) · Immunoglobulin Light-chain Amyloidosis (3) · Aortic Valve Stenosis (2) · Heart Diseases (2) · Heart Failure (1) · Heart Failure, Diastolic (1) · Median Neuropathy (1)
Metabolic Diseases (27) · Nutritional and Metabolic Diseases (27) · Proteostasis Deficiencies (27) · Nervous System Diseases (18) · Neuromuscular Diseases (17) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Metabolism, Inborn Errors (16) · Peripheral Nervous System Diseases (16) · Neurodegenerative Diseases (14) · Heredodegenerative Disorders, Nervous System (13) · Amyloidosis (12) · Amyloidosis, Familial (12) · Amyloid Neuropathies (11) · Cardiovascular Diseases (11)
attruby (3) · patisiran (3) · Biopsy (2) · Blood Specimen Collection (2) · Doxycycline (2) · Amyloid (1) · Echocardiography (1) · Hematologic Tests (1) · Observation (1) · Tablets (1) · Technetium Tc 99m Pyrophosphate (1) · Tolcapone (1) · Ursodeoxycholic Acid (1) · tafamidis (1) · ursodoxicoltaurine (1)
The Cleveland Clinic (4) · AstraZeneca (3) · Eidos Therapeutics, a BridgeBio company (3) · Alnylam Pharmaceuticals (2) · CENTOGENE GmbH Rostock (2) · Steen Hvitfeldt Poulsen (2) · Assistance Publique Hopitaux De Marseille (1) · Austin Neuromuscular Center (1) · Boston University (1) · Centre Hospitalier Universitaire de Nīmes (1) · Clinique Saint Jean, France (1) · Columbia University (1) · Fondazione IRCCS Policlinico San Matteo di Pavia (1) · John Paul II Hospital, Krakow (1) · Katarzyna Holcman (1)
United States (19) · France (10) · Germany (9) · Italy (7) · Spain (7) · United Kingdom (7) · Canada (6) · Portugal (6) · Denmark (5) · Sweden (5) · Belgium (4) · Brazil (4) · Netherlands (4) · Poland (4) · South Korea (4)
Mazen A Hanna, MD (3) · Arndt Rolfs, Prof. (1) · Edward Miller, MD (1) · Elisabete Martins, MD, PhD (1) · Erwan Donal (1) · Giampaolo Merlini, MD (1) · Hanna K Gaggin, MD (1) · John L Berk, MD (1) · Mathew Maurer, MD (1) · Mazen Hanna, MD (1) · Myriam Alexander, PHD (1) · Ole B Suhr, MD PhD Prof (1)
PMID 18579156, PMID 22056963, PMID 23193944, PMID 25091367, PMID 25372741, PMID 26870387, PMID 27386769, PMID 28384285, PMID 29633228, PMID 30010028, PMID 30336828, PMID 30810593, PMID 30938420, PMID 31163298, PMID 31353964, PMID 31726319, PMID 32169556, PMID 33725199, PMID 34024024, PMID 34148211, PMID 34331265, PMID 34729526, PMID 35188911, PMID 35213769, PMID 35451899, PMID 35583798, PMID 35717381, PMID 35730447, PMID 35875890, PMID 37422895, PMID 37523143, PMID 37639169, PMID 37946256, PMID 38117424, PMID 38197816, PMID 38241252, PMID 38512694, PMID 38909877, PMID 39105117, PMID 39537877, PMID 39878480, PMID 40074465, PMID 40108078, PMID 40185370, PMID 40398971, PMID 40439628, PMID 40586114, PMID 40632476, PMID 40643127, PMID 40750720
2018 (3), 2023 (1)