3 completed, 3 recruiting, 2 active not recruiting, 2 approved for marketing. 4 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 4 | Completed 3, Recruiting 1 |
Follow Amyloidosis, Hereditary: new trials by email Literature Review of Amyloidosis, Hereditary trials Research Report on Amyloidosis, Hereditary
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 |
|---|---|---|---|---|---|---|
| Southeastern ATTR Amyloidosis Consortium: SEATTRAC Family Registry Registry - Amyloidosis, Hereditary | Not yet recruiting | Virginia Commonwealth University | 1000 | 2026-09-01 | 2026-08-12 | |
| 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 |
| ATTR Amyloid Cardiomyopathy: Characterization of Extracellular Vesicles as Potential Disease Stratifiers and Prognostic Biomarkers Wild-type ATTR Amyloidosis, Amyloidosis Cardiac | Recruiting | University of Sao Paulo General Hospital | 70 | 2024-10-18 | 2026-01-02 | |
| 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 | |
| Prospective Evaluation of NfL as a Biomarker in ATTRv Amyloidosis, Hereditary | Active not recruiting | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 40 | 2023-09-01 | 2025-08-14 | |
| Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers Amyloidosis, Hereditary, Amyloidosis Cardiac | Recruiting | University of Texas Southwestern Medical Center | 500 | 2022-11-21 | 2026-08-31 | |
| HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) Patisiran, Vutrisiran - Amyloidosis, Hereditary, Transthyretin Amyloidosis | Phase 3 | Completed | Alnylam Pharmaceuticals | 164 | 2019-02-14 | 2026-01-12 |
| ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl) - Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary | Phase 3 | Completed | Alnylam Pharmaceuticals | 206 | 2014-12-01 | 2018-07-18 |
| APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) - TTR-mediated Amyloidosis, Amyloidosis, Hereditary | Phase 3 | Completed | Alnylam Pharmaceuticals | 225 | 2013-11-01 | 2024-04-22 |
| ATTR Expanded Access Program (EAP) by Ionis Inotersen - Amyloidosis, Hereditary | Approved for marketing | Ionis Pharmaceuticals, Inc. | 2019-08-05 | |||
| Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR) patisiran (ALN-TTR02) - TTR-mediated Amyloidosis, Amyloidosis, Hereditary | Approved for marketing | Alnylam Pharmaceuticals | 2024-05-20 |
Amyloidosis, Familial (11) · Amyloidosis, Hereditary, Transthyretin-Related (7) · Amyloid Neuropathies, Familial (6) · Amyloid Neuropathies (4) · Amyloidosis (4) · Alzheimer Disease (1) · Cardiomyopathies (1) · Disease (1) · Disease Progression (1) · Genetic Diseases, Inborn (1) · Heart Failure (1) · Heredodegenerative Disorders, Nervous System (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1) · Nervous System Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Nutritional and Metabolic Diseases (11) · Proteostasis Deficiencies (11) · Genetic Diseases, Inborn (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Amyloidosis (7) · Heredodegenerative Disorders, Nervous System (5) · Nervous System Diseases (5) · Neurodegenerative Diseases (5) · Neuromuscular Diseases (5) · Peripheral Nervous System Diseases (5) · Amyloid Neuropathies (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2)
patisiran (3) · Inotersen (1) · NTLA-2001 (1) · Registries (1) · Saline Solution (1) · revusiran (1)
Alnylam Pharmaceuticals (4) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (1) · Intellia Therapeutics (1) · Ionis Pharmaceuticals, Inc. (1) · Rennes University Hospital (1) · University of Sao Paulo General Hospital (1) · University of Texas Southwestern Medical Center (1) · Virginia Commonwealth University (1)
United States (5) · Brazil (4) · France (4) · Italy (4) · Argentina (3) · Canada (3) · Germany (3) · Mexico (3) · Spain (3) · Sweden (3) · Taiwan (3) · United Kingdom (3) · Australia (2) · Belgium (2) · Bulgaria (2)
Erwan Donal (1) · Jared Gollob (1) · Jared Gollob, MD (1) · Justin L Grodin, MD MPH (1) · Keyur Shah, MD (1) · Marco Luigetti (1)
PMID 28893208, PMID 29972753, PMID 30586695, PMID 30878017, PMID 31215818, PMID 31322739, PMID 32062791, PMID 35875890, PMID 36120830, PMID 37523143, PMID 38512694, PMID 38548524, PMID 42290201
2018 (1), 2023 (1), R01HL160892 (1)