11 completed, 1 approved for marketing, 1 no longer available. 5 Phase 3, 2 Phase 1, 2 Phase 2, 1 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 5 | Completed 5 |
| Phase 1 | 2 | Completed 2 |
| Phase 2 | 2 | Completed 2 |
| Early Phase 1 | 1 | Completed 1 |
Follow patisiran: new trials by email Literature Review of patisiran trials Research Report on patisiran
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 |
|---|---|---|---|---|---|---|
| Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy patisiran - Polyneuropathies, Wild Type ATTR Amyloidosis | Early Phase 1 | Completed | Austin Neuromuscular Center | 10 | 2022-08-03 | 2026-05-22 |
| A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation Patisiran - Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy | Completed | Alnylam Pharmaceuticals | 67 | 2019-12-18 | 2022-06-06 | |
| APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) Placebo, Patisiran - Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Phase 3 | Completed | Alnylam Pharmaceuticals | 360 | 2019-09-04 | 2026-04-20 |
| Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant Patisiran - Amyloidosis, Familial, Transthyretin Amyloidosis | Phase 3 | Completed | Alnylam Pharmaceuticals | 24 | 2019-03-27 | 2024-04-22 |
| 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 |
| The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran) Patisiran - Amyloidosis | Phase 3 | Completed | Alnylam Pharmaceuticals | 211 | 2015-07-16 | 2023-12-06 |
| A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl) - Transthyretin (TTR)-Mediated Amyloidosis | Phase 1 | Completed | Alnylam Pharmaceuticals | 12 | 2014-01-01 | 2015-05-25 |
| 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 |
| 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 | |||
| Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy Patisiran - Transthyretin-mediated Amyloidosis With Cardiomyopathy, ATTR Amyloidosis With Cardiomyopathy | No longer available | Alnylam Pharmaceuticals | 2024-02-16 |
Amyloidosis, Hereditary, Transthyretin-Related (7) · Amyloidosis (6) · Amyloid Neuropathies, Familial (5) · Amyloidosis, Familial (5) · Amyloid Neuropathies (3) · Cardiomyopathies (2) · Polyneuropathies (2) · Genetic Diseases, Inborn (1) · Heredodegenerative Disorders, Nervous System (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1) · Nervous System Diseases (1) · Neurodegenerative Diseases (1) · Neuromuscular Diseases (1) · Peripheral Nervous System Diseases (1)
Alnylam Pharmaceuticals (12) · Austin Neuromuscular Center (1)
United States (9) · France (7) · Portugal (7) · Sweden (7) · United Kingdom (7) · Brazil (6) · Germany (6) · Spain (6) · Italy (5) · Argentina (4) · Australia (4) · Bulgaria (4) · Japan (4) · Mexico (4) · Netherlands (4)
Jared Gollob, MD (4) · Jared Gollob (1)
PMID 38548524, PMID 23984729, PMID 26338094, PMID 28893208, PMID 29972753, PMID 30586695, PMID 30878017, PMID 31215818, PMID 31322739, PMID 32641071, PMID 33212063, PMID 35188911, PMID 35213769, PMID 35875890, PMID 36120830, PMID 37523143, PMID 37639169, PMID 37888916, PMID 38512694, PMID 39804640, PMID 40243852, PMID 42290201
2018 (2), 2014 (1), 2019 (1), 2023 (1)