7 completed, 1 approved for marketing. 4 Phase 2, 2 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 4 |
| Phase 1 | 2 | Completed 2 |
| Phase 3 | 1 | Completed 1 |
Follow TTR-mediated Amyloidosis: new trials by email Literature Review of TTR-mediated Amyloidosis trials Research Report on TTR-mediated 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 Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis Revusiran (ALN-TTRSC) - TTR-mediated Amyloidosis | Phase 2 | Completed | Alnylam Pharmaceuticals | 25 | 2014-10-01 | 2020-07-09 |
| Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis ALN-TTRSC (revusiran) for subcutaneous administration - TTR-mediated Amyloidosis | Phase 2 | Completed | Alnylam Pharmaceuticals | 26 | 2013-12-01 | 2023-03-20 |
| 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 |
| The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran) ALN-TTR02 (patisiran) administered by intravenous (IV) infusion - TTR-mediated Amyloidosis | Phase 2 | Completed | Alnylam Pharmaceuticals | 27 | 2013-10-01 | 2024-04-19 |
| A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers ALN-TTRSC (revusiran), Sterile Normal Saline (0.9% NaCl) - TTR-mediated Amyloidosis | Phase 1 | Completed | Alnylam Pharmaceuticals | 85 | 2013-03-01 | 2016-02-05 |
| Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis Patisiran - TTR-mediated Amyloidosis | Phase 2 | Completed | Alnylam Pharmaceuticals | 29 | 2012-05-01 | 2024-04-19 |
| Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects ALN-TTR02, Sterile Normal Saline (0.9% NaCl) - TTR-mediated Amyloidosis | Phase 1 | Completed | Alnylam Pharmaceuticals | 17 | 2012-03-01 | 2013-01-15 |
| 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 |
Amyloid Neuropathies (2) · Amyloid Neuropathies, Familial (2) · Amyloidosis (2) · Amyloidosis, Familial (2) · Amyloidosis, Hereditary, Transthyretin-Related (2) · Alzheimer Disease (1) · Cardiomyopathies (1) · Heart Failure (1)
Nervous System Diseases (3) · Neurodegenerative Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Heredodegenerative Disorders, Nervous System (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Neuromuscular Diseases (2) · Nutritional and Metabolic Diseases (2) · Peripheral Nervous System Diseases (2) · Proteostasis Deficiencies (2) · Brain Diseases (1) · Cardiovascular Diseases (1) · Central Nervous System Diseases (1) · Dementia (1)
United Kingdom (5) · United States (5) · Brazil (3) · France (3) · Germany (3) · Portugal (3) · Spain (3) · Sweden (3) · Canada (2) · Argentina (1) · Australia (1) · Bulgaria (1) · Cyprus (1) · Italy (1) · Japan (1)
Jared Gollob, MD (6) · Jared Gollob (1)
PMID 23984729, PMID 26338094, PMID 28893208, PMID 29972753, PMID 30586695, PMID 30878017, PMID 31215818, PMID 31322739, PMID 32641071, PMID 36120830, PMID 38548524