3 completed, 1 recruiting. 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
Amyloidosis, Familial (4) · Amyloidosis, Hereditary, Transthyretin-Related (4) · Amyloid Neuropathies (3) · Amyloid Neuropathies, Familial (3) · Amyloidosis (2) · Alzheimer Disease (1) · Cardiomyopathies (1) · Genetic Diseases, Inborn (1) · Heart Failure (1) · Heredodegenerative Disorders, Nervous System (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1) · Nervous System Diseases (1) · Neurodegenerative Diseases (1) · Neuromuscular Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Nutritional and Metabolic Diseases (4) · Proteostasis Deficiencies (4) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Amyloidosis (2) · Heredodegenerative Disorders, Nervous System (2) · Nervous System Diseases (2) · Neurodegenerative Diseases (2) · Neuromuscular Diseases (2) · Peripheral Nervous System Diseases (2) · Brain Diseases (1) · Cardiovascular Diseases (1) · Central Nervous System Diseases (1)
patisiran (2) · NTLA-2001 (1) · Saline Solution (1) · revusiran (1)
Alnylam Pharmaceuticals (3) · Intellia Therapeutics (1)
Argentina (3) · Brazil (3) · Canada (3) · France (3) · Germany (3) · Italy (3) · Mexico (3) · Spain (3) · Sweden (3) · Taiwan (3) · United Kingdom (3) · United States (3) · Australia (2) · Belgium (2) · Bulgaria (2)
Jared Gollob (1) · Jared Gollob, MD (1)
PMID 28893208, PMID 29972753, PMID 30586695, PMID 30878017, PMID 31322739, PMID 32062791, PMID 35875890, PMID 36120830, PMID 37523143, PMID 38512694, PMID 38548524, PMID 42290201
2018 (1), 2023 (1)