1 approved for marketing, 1 completed. 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 1 | Completed 1 |
Follow Familial Amyloid Polyneuropathies: new trials by email Literature Review of Familial Amyloid Polyneuropathies trials Research Report on Familial Amyloid Polyneuropathies
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Heredodegenerative Disorders, Nervous System (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Nervous System Diseases (2) · Neurodegenerative Diseases (2) · Neuromuscular Diseases (2) · Nutritional and Metabolic Diseases (2) · Peripheral Nervous System Diseases (2) · Proteostasis Deficiencies (2)
patisiran (2)
Argentina (1) · Australia (1) · Brazil (1) · Bulgaria (1) · Canada (1) · Cyprus (1) · France (1) · Germany (1) · Italy (1) · Japan (1) · Malaysia (1) · Mexico (1) · Netherlands (1) · Portugal (1) · South Korea (1)
Jared Gollob (1)
PMID 28893208, PMID 29972753, PMID 30586695, PMID 30878017, PMID 31215818, PMID 31322739, PMID 36120830, PMID 38548524