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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) 20 Mg Or 80 Mg In Comparison To Placebo In Subjects Diagnosed With Transthyretin Cardiomyopathy (TTR-CM) (ATTR-ACT)
Rochester, Minn.
This Phase 3 study will investigate the efficacy, safety and tolerability of an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules compared to placebo in subjects with either transthyretin genetic variants or wild-type transthyretin resulting in amyloid cardiomyopathy.
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A Phase 1, Open-label, Dose Escalation Study of Intravenous PRX004 in Subjects With Amyloid Transthyretin (ATTR) Amyloidosis
Rochester, Minn.
A Phase 1, open-label, 3+3 dose escalation component to determine the safety, tolerability, PK, PD, and MTD of IV PRX004 when given as a single agent to up to 36 subjects with hATTR amyloidosis An expansion component in up to 2 anticipated PRX004 RP2D cohorts selected from dose escalation (up to an additional 3 subjects in each expanded cohort for up to 6 evaluable subjects total in each expanded cohort; an evaluable subject is defined as a subject who has completed the first 28 days following the first administration of PRX004 or who discontinued within 28 days due to study drug related toxicity). Subjects with wtATTR amyloidosis may be enrolled as part of the expansion component.
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A Phase 2, Randomized, Placebo-controlled, Dose-ranging Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AG10 in Patients With Symptomatic Transthyretin Amyloid Cardiomyopathy
Rochester, Minn.
This prospective, randomized, multicenter, double-blind, parallel group, placebo-controlled, dose-ranging study will evaluate the safety, tolerability, PK and PD of AG10 compared to placebo administered on a background of stable heart failure therapy. Screening and randomization will be followed by a 28-day blinded, placebo-controlled treatment period.
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A Phase 3 Multicenter Open-Label Study to Evaluate the Safety of Daily Oral Dosing of Tafamidis Meglumine (PF-06291826-83) 20 mg or 80 mg or Tafamidis (PF-06291826-00) 61 mg in Subjects Diagnosed with Transthyretin Cardiomyopathy (ATTR-CM)
Rochester, Minn.
The purpose of this study is to evaluate the long-term safety of tafamidis meglumine for treating patients who have either the genetic variants or wild-type transthyretin, resulting in amyloid heart muscle disease.
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A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
Rochester, Minn.
The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy. Dosing has been discontinued; patients are being followed-up for safety.
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A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTRIBUTE-CM) (ATTRIBUTE-CM)
Rochester, Minn.
The purpose of this study is to evaluate AG10 800 mg compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
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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)
Rochester, Minn.
The purpose of this study is to assess safety and tolerability of Acoramidis in participants with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM).
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An Open-Label Extension and Safety Monitoring Study of Patients with Symptomatic Transthyretin Cardiomyopathy Who Have Completed the Phase II Study AG10-201
Rochester, Minn.
The purpose of this study is to
The primary objective of this study is to evaluate the long-term safety and tolerability of AG10 administered to adult patients with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM) in patients who have completed the study AG10-201.
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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 Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Rochester, Minn.
The purpose of this study is to evaluate the effectiveness and safety of patisiran in participants with Transthyretin Amyloidosis With Cardiomyopathy.
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Efficacy and Safety of NNC6019-0001 at Two Dose Levels in Participants With Transthyretin Amyloid Cardiomyopathy (ATTR CM)
Rochester, Minn.,
Scottsdale/Phoenix, Ariz.,
Jacksonville, Fla.
The purpose of this study is test the effectiveness and safety of NNC6019-0001 to determine if it can reduce the symptoms of a heart disease due to TTR (Transthyretin ) amyloidosis, such as heart failure.
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HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Rochester, Minn.
The purpose of this study is to evaluate the effectiveness and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis).
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HELIOS-B: A Phase 3, Randomized, Doubleblind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
Rochester, Minn.
The purpose of this study is to evaluate vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy.
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