42 completed, 9 recruiting, 6 active not recruiting, 4 not yet recruiting. 74 Phase 3, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 74 | Completed 42, Recruiting 9, Active not recruiting 6, Not yet recruiting 4, Terminated 4, Unknown 4, Withdrawn 3, Enrolling by invitation 2 |
| Phase 2 | 5 | Completed 5 |
Follow Amyloidosis: new trials by email Literature Review of Amyloidosis trials Research Report on 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 |
|---|---|---|---|---|---|---|
| A Randomized Phase 3 Study to Evaluate the Efficacy and Safety of NXC-201 Compared With Daratumumab With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) in Newly Diagnosed Systemic AL Amyloidosis NXC-201, Dara-CyBorD - Light Chain (AL) Amyloidosis | Phase 3 | Not yet recruiting | Nexcella Inc. | 260 | 2027-01-01 | 2026-07-16 |
| Linvoseltamab Versus Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone (Dara-Vcd) in Adult Participants With Newly Diagnosed Systemic Light Chain Amyloidosis Linvoseltamab, Daratumumab, Bortezomib - Newly Diagnosed Amyloidosis, Amyloidosis Stage I-IIIA | Phase 3 | Not yet recruiting | Regeneron Pharmaceuticals | 382 | 2026-12-18 | 2026-10-28 |
| Study to Evaluate Change in Disease Activity and Adverse Events of Subcutaneous Etentamig Compared With Daratumumab Plus Cyclophosphamide Plus Bortezomib Plus Dexamethasone (Dara-CyBorD) in Adults With Amyloid Light Chain (AL) Amyloidosis Etentamig, Daratumumab, Cyclophosphamide - Amyloid Light Chain (AL) Amyloidosis | Phase 3 | Not yet recruiting | AbbVie | 370 | 2026-11-29 | 2026-08-28 |
| 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 |
| [64Cu]FBP8 PET for Early Detection of Intracardiac Thrombus in Amyloid Cardiomyopathy [64Cu]FBP8 PET/MR - Cardiac Amyloidosis | Phase 3 | Recruiting | Brigham and Women's Hospital | 20 | 2026-07-07 | 2026-07-21 |
| A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis Coagadex(R) - Acquired Factor X Deficiency | Phase 3 | Withdrawn | Kedrion S.p.A. | 2026-06-01 | 2026-06-29 | |
| TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy Nucresiran, Vutrisiran - Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN | Phase 3 | Recruiting | Alnylam Pharmaceuticals | 125 | 2026-02-03 | 2026-10-08 |
| Jiedu Huayu Oral Prescription in the Treatment of Intracranial Hemorrhage Associated With Cerebral Amyloid Angiopathy Jiedu Huayu Oral Prescription, Placebo - Cerebral Amyloid Angiopathy | Phase 3 | Recruiting | Beijing Tiantan Hospital | 436 | 2026-01-30 | 2026-09-30 |
| A Study to Evaluate the Safety and Efficacy of SCTC21C in Combination With Bortezomib, Cyclophosphamide, and Dexamethasone in Patients With Newly Diagnosed Systemic Light-Chain Amyloidosis (NDSLCA) SCTC21C, Bortezomib, Dexamethasone - Amyloidosis | Phase 3 | Recruiting | Sinocelltech Ltd. | 90 | 2026-01-08 | 2026-02-05 |
| TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Nucresiran, Sterile Normal Saline (0.9% NaCl) - Transthyretin Amyloidosis With Cardiomyopathy | Phase 3 | Recruiting | Alnylam Pharmaceuticals | 1750 | 2025-07-02 | 2026-10-20 |
| 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 |
| REVEAL - Research With I-124 EVuzamitide to Elucidate Cardiac AmyLoidosis I-124 evuzamitide - Cardiac Amyloidosis | Phase 3 | Completed | Sharmila Dorbala | 204 | 2025-01-14 | 2026-04-30 |
| 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 |
| Comparing Dara-VCD Chemotherapy Plus Stem Cell Transplant to Dara-VCD Chemotherapy Alone for People Who Have Newly Diagnosed AL Amyloidosis Autologous Hematopoietic Stem Cell Transplantation, Biopsy, Biospecimen Collection - AL Amyloidosis | Phase 3 | Recruiting | SWOG Cancer Research Network | 338 | 2024-07-01 | 2026-05-06 |
| Study of ALXN2220 Versus Placebo in Adults With ATTR-CM ALXN2220, Placebo - Transthyretin Amyloid Cardiomyopathy | Phase 3 | Active not recruiting | Alexion Pharmaceuticals, Inc. | 1182 | 2024-01-11 | 2026-10-22 |
| MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) NTLA-2001, Placebo - Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Phase 3 | Recruiting | Intellia Therapeutics | 1200 | 2023-12-13 | 2026-03-27 |
| Probiotic Supplementation Reduces Gastrointestinal Symptoms During the Therapy and Improves Therapeutic Response in AL Amyloidosis Live Combined Bacillus Subtilis and Enterococcus Faecium Enteric-coated Capsules combined with Bortezomib+Dexamethasone/Bortezomib+Dexamethasone+Daratumumab/single Daratumumab, Bortezomib+Dexamethasone/Bortezomib+Dexamethasone+Daratumumab/single Daratumumab - Immunoglobulin Light-chain Amyloidosis | Phase 3 | Unknown | Air Force Military Medical University, China | 116 | 2023-03-31 | 2023-03-20 |
| Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis [18F]florbetaben - Cardiac Amyloidosis, AL Amyloidosis | Phase 3 | Completed | Lantheus Germany GmbH | 244 | 2023-01-13 | 2026-10-26 |
| Molecular Imaging of Myocardial Fibrosis in Cardiac Amyloidosis [68Ga]CBP8 PET/MRI - Cardiac Amyloidosis | Phase 3 | Active not recruiting | Brigham and Women's Hospital | 18 | 2023-01-03 | 2026-07-06 |
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Immunoglobulin Light-chain Amyloidosis (29) · Amyloid Neuropathies, Familial (22) · Multiple Myeloma (15) · Amyloidosis, Hereditary, Transthyretin-Related (14) · Hodgkin Disease (13) · Immunoblastic Lymphadenopathy (13) · Leukemia, Lymphocytic, Chronic, B-Cell (13) · Lymphoma, Follicular (13) · Lymphoma, Large B-Cell, Diffuse (13) · Lymphoma, Large-Cell, Anaplastic (13) · Lymphoma, Large-Cell, Immunoblastic (13) · Lymphoma, Mantle-Cell (13) · Lymphoma, Non-Hodgkin (13) · Lymphoma, T-Cell, Cutaneous (13)
Nutritional and Metabolic Diseases (74) · Proteostasis Deficiencies (74) · Metabolic Diseases (73) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (35) · Cardiovascular Diseases (31) · Immune System Diseases (30) · Nervous System Diseases (30) · Genetic Diseases, Inborn (29) · Immunoproliferative Disorders (29) · Neoplasms by Histologic Type (29) · Paraproteinemias (27) · Neoplasms (26) · Metabolism, Inborn Errors (25) · Lymphoproliferative Disorders (24)
Dexamethasone (12) · Bortezomib (10) · Melphalan (9) · Cyclophosphamide (8) · attruby (5) · patisiran (5) · tafamidis (5) · Filgrastim (4) · daratumumab (4) · Calcium Dobesilate (3) · Inotersen (3) · eplontersen (3) · Clinical Protocols (2) · Darbepoetin alfa (2) · Early Intervention, Educational (2)
Alnylam Pharmaceuticals (10) · Eidos Therapeutics, a BridgeBio company (6) · Ionis Pharmaceuticals, Inc. (5) · Pfizer (5) · Alexion Pharmaceuticals, Inc. (3) · National Taiwan University Hospital (3) · Alliance for Clinical Trials in Oncology (2) · Brigham and Women's Hospital (2) · Intellia Therapeutics (2) · Jonsson Comprehensive Cancer Center (2) · Mayo Clinic (2) · Memorial Sloan Kettering Cancer Center (2) · Prothena Biosciences Ltd. (2) · AbbVie (1) · Air Force Military Medical University, China (1)
United States (52) · United Kingdom (33) · France (32) · Italy (32) · Spain (32) · Germany (29) · Brazil (26) · Canada (24) · Sweden (24) · Australia (22) · Belgium (22) · Netherlands (22) · Portugal (22) · Argentina (20) · Japan (19)
Jeff Packman (2) · John A. Glaspy, MD, MPH (2) · Mark McGovern, RN, CCRN (2) · Scott Swenson, MD (2) · Andrew Stephens, MD, PhD (1) · Angela Dispenzieri, M.D. (1) · Ann Traynor, MD (1) · Arnaud Jaccard, MD (1) · Barry J.A. Laird, MD (1) · Charles L. Loprinzi, MD (1) · David J. Straus, MD (1)
PMID 38065307, PMID 27494299, PMID 30558645, PMID 31353964, PMID 32107748, PMID 32524297, PMID 33070419, PMID 34355354, PMID 34923848, PMID 35799473, PMID 36715498, PMID 37366170, PMID 37434378, PMID 37943223, PMID 38548524, PMID 38932583, PMID 39213194, PMID 40488446, PMID 41205147, PMID 41913562, PMID 41937511, PMID 42212672, PMID 42389794, PMID 42658466, PMID 42669065, PMID 15459300, PMID 16710026, PMID 17397698, PMID 17554116, PMID 17855669, PMID 20085941, PMID 22480131, PMID 23974642, PMID 24368466, PMID 24386911, PMID 26700120, PMID 26700123, PMID 27020837, PMID 28393570, PMID 28550039, PMID 28611125, PMID 28893208, PMID 29115008, PMID 29972753, PMID 29972757, PMID 30145929, PMID 30169969, PMID 30219500, PMID 30586695, PMID 30878017
2018 (4), 2019 (3), 2016 (2), 2023 (2), P30CA015083 (2), 2014 (1), 2025 (1), P30CA068485 (1), R01CA127655 (1), R01NS051306 (1), U10CA021115 (1), U10CA180888 (1)