An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)11/21/2024 - 11/21/2027 (PI)
Alnylam Pharmaceuticals, Inc.
ALXN2220-ATTRCM-301, D6810C00001
A Phase 3, Randomized, Double-Blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants wit05/03/2024 - 05/03/2027 (PI)
Alexion Pharmaceuticals, Inc.
An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) ION682884-CS1212/20/2023 - 12/20/2026 (PI)
Ionis Pharmaceuticals, Inc.
ALN-TTR02-013: ConTTRibute: A Global Observational Multicenter Long-Term Study of Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR amyloidosis)09/22/2021 - 09/22/2026 (PI)
Alnylam Pharmaceuticals, Inc.
Protocol ALN-TTR02-014: Expanded Access Protocol to provide Patisiran to Patients with Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) with Cardiomyopathy06/16/2022 - 06/16/2025 (PI)
Alnylam Pharmaceuticals, Inc.
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients with Familial Amyloidollo Polyneuropathy Who Have02/04/2014 - 02/28/2025 (PI)
Alnylam Pharmaceuticals, Inc.
Protocol ION-682884-CS2: A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomy03/20/2020 - 11/19/2024 (Subcontract PI)
Pharmaceutical Research Associates, Inc. IonisPharmaceuticals
Protocol # ION-682844-CS3: A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy11/15/2019 - 09/16/2024 (Subcontract PI)
Pharmaceutical Research Associates, Inc. IonisPharmaceuticals
"ALN-TTR02-011, APOLLO-B: A Phase 3, Randomized,
Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of
Patisiran in Patients with Transthyretin Amyloidosis with Car09/12/2019 - 09/12/2024 (PI)
Alnylam Pharmaceuticals, Inc.
[ION-682884-CS13]: An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy09/28/2021 - 08/03/2024 (PI)
Ionis Pharmaceuticals, Inc.
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An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)10/18/2021 - 07/20/2024 (PI)
Eidos Therapeutics, Inc., A BridgeBio Company
ALN-TTRSC02, HELIOS-B: A Phase 3, Randomized, Double blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients with Transthyretin Amyloidosis with Cara12/04/2019 - 07/20/2024 (PI)
Alnylam Pharmaceuticals, Inc.
Phase 3 Multicenter, Open-label Study to Evaluate the Safety of Daily Oral Dosing of Tafamidis Meglumine (PF-06291826) 20 MG or 80 MG in subjects diagnosed with transthyrtin cardiomyothy.04/04/2017 - 04/03/2024 (PI)
Pfizer, Inc.
"A Phase 3, Randomized, Double-Blind, Placebo-controlled Study of the Efficacy and Safety of AG10 in Subjects with Symptomatic Transthyretin Amyloid Cardiomyopathy"10/04/2019 - 02/05/2024 (PI)
Eidos Therapeutics, Inc., A BridgeBio Company
HELIOS-A: A Phase 3 Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with hATTR Amyloidosis03/28/2019 - 11/14/2023 (PI)
Alnylam Pharmaceuticals, Inc.
Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Clinical Study with03/29/2016 - 03/28/2023 (PI)
Alnylam Pharmaceuticals, Inc.
An Open-Label, Investigator Study to Evaluate the Short-term (4 Weeks) Effects of TOLCAPONE on Transthyretin Stability in Subjects with Leptomeningeal TTR Amyloidosis (ATTR) with and without CNS…09/14/2018 - 08/12/2022 (PI)
Corino Therapeutics
An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy09/30/2014 - 04/15/2022 (PI)
Ionis Pharmaceuticals, Inc.
A study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease03/27/2019 - 03/26/2022 (Co-Investigator)
PI:
Andrea Havasi, MDAlnylam Pharmaceuticals, Inc.
Expanded Access Protocol of Patisiran for Patients with Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polynenropathy10/31/2016 - 10/30/2021 (PI)
Alnylam Pharmaceuticals, Inc.
Protocol #ISIS 420915-CS4, A Non-Interventional Study to Monitor the Survival Status of Patients That Discontinued from ISIS 420915-CS2 or ISIS 420915-CS305/14/2018 - 05/13/2021 (PI)
Ionis Pharmaceuticals, Inc.
Protocol #CS5 - Expanded Access Program for Inotersen (ISIS 420915) in Patients with Hereditary Transthyretin Amyloidosis (hATTR)04/17/2018 - 04/16/2021 (PI)
Ionis Pharmaceuticals, Inc.
Protocol # EIDOS AG10-101, Assessment of the TTR Binding and Stabilizing Properties of AG10 to Different Variant TTRs in the Sera or Plasma from ATTR Patients or Relatives with Different TTR Genotypes03/05/2018 - 03/04/2021 (PI)
Eidos Therapeutics, Inc., A BridgeBio Company
Protocol # AG10-201, A Phase 2, Randomized, Placebo-controlled, Dose-ranging Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AG10 in Patients with Symptomatic Transthyretin03/05/2018 - 03/04/2021 (PI)
Eidos Therapeutics, Inc., A BridgeBio Company
A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlle Study to Evaluate the Efficacy and Safety of ALN-TTRSC in Patients with Transthyretin Mediated Familial Amyloidotic07/28/2015 - 07/28/2020 (PI)
Alnylam Pharmaceuticals, Inc.
Treatment Use of Eprodisate (Klacta) In a Patient Diagnosed with AA Amyloidosis02/16/2016 - 02/15/2019 (PI)
A.T. Development Switzerland Sarl
A Multicenter, International, Phase 3, Double-Blind, Placebo-Controlled, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of Daily Oral Dosing of Tafamidis Meglumine (PF-06291826)10/17/2014 - 05/31/2018 (PI)
Pfizer, Inc.
BU-Alnylam Diflunisal Research Collaboration10/01/2015 - 10/15/2017 (PI)
Alnylam Pharmaceuticals, Inc.
APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of ALN-TTR02 in Transthyretin (TTR)-Mediated Polyneuropathy07/01/2014 - 09/30/2017 (PI)
Alnylam Pharmaceuticals, Inc.
A phase 2, open-label, multi-dose, dose escalation trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous infusions of ALN-TTR02 in patients with TTR Amyloid08/27/2013 - 08/31/2017 (PI)
Alnylam Pharmaceuticals, Inc.
International Randomized Double-Blind, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of KIACTA in Preventing renal Function Decline in Patients with AA amyloidosis08/01/2011 - 07/31/2017 (PI)
PPD Development LLC
A phase 2/3 randomized, double-blind, placebo controlled study to assess the efficacy and safety of Isis 420915 In Patients with Amyloid Polyneuropathy03/01/2013 - 02/28/2017 (PI)
Ionis Pharmaceuticals, Inc.
A Randomised, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of GSK2998728 in Subjects with Transthyretin Amyloid Cardiomyopathy, FAC20465012/17/2015 - 12/31/2016 (PI)
GlaxoSmithKline LLC
A Natural History Study to Characterize Circulating Wild Type and Mutant Transthyretin (TTR) Levels in Adult Carriers of TTR Gene Mutations09/29/2009 - 09/30/2015 (PI)
Alnylam Pharmaceuticals, Inc.
Effect of Diflunisal on Familial Amyloidosis09/15/2005 - 06/30/2013 (PI)
NIH/National Institute of Neurological Disorders & Stroke5R01NS051306-05
International Randomized Double-Blind, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of KIACTA in Preventing renal Function Decline in Patients with AA amyloidosis08/01/2011 - 07/31/2012 (PI)
PPD Development LLC