Clinical Trials on Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Total 119 results

Alnylam Pharmaceuticals

Completed

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Amyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac Amyloidosis

United States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom
Alnylam Pharmaceuticals

Completed

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

ATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid Neuropathies

France, Germany, Portugal, United Kingdom, Sweden, Spain
Pfizer

Completed

Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers

Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) | Transthyretin Cardiomyopathy (TTR-CM) | Familial Amyloid Cardiomyopathy | Senile Systemic Amyloidosis (SSA)
Alnylam Pharmaceuticals

Completed

A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers

Transthyretin (TTR)-Mediated Amyloidosis

United Kingdom
Alnylam Pharmaceuticals

No longer available

Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

Transthyretin-mediated Amyloidosis With Cardiomyopathy | ATTR Amyloidosis With Cardiomyopathy

United States
CENTOGENE GmbH Rostock
Alnylam Pharmaceuticals

Recruiting

TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects (TRAMmoniTTR)

Cardiomyopathies | Polyneuropathies | Transthyretin Amyloidosis | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Germany, Austria, Switzerland
Alnylam Pharmaceuticals

Completed

DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis

Familial Amyloidotic Cardiomyopathy (FAC)

United States, France, Spain, United Kingdom, Belgium, Brazil
Alnylam Pharmaceuticals

Completed

Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis

Transthyretin Mediated Amyloidosis (ATTR)

United Kingdom, France, Portugal, Sweden
University of Texas Southwestern Medical Center
National Heart, Lung, and Blood Institute (NHLBI); Columbia University; The Cleveland... and other collaborators

Recruiting

Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

Amyloidosis, Familial | Amyloidosis, Hereditary | Amyloidosis Cardiac | Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin Gene Mutation

United States
Ionis Pharmaceuticals, Inc.
AstraZeneca

Recruiting

An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

United States, Austria, Belgium, Canada, France, Germany, Israel, Italy, Portugal, Spain, Sweden, United Kingdom, Australia, Greece
Ionis Pharmaceuticals, Inc.

Active, not recruiting

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

United States, Australia, Spain, Canada, Germany, Japan, Italy, Israel, Argentina, France, Portugal, Austria, Brazil, Greece, United Kingdom, Czechia, Sweden, Belgium, Denmark, Poland, Puerto Rico
Intellia Therapeutics

Recruiting

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

United Kingdom, New Zealand, Sweden
Intellia Therapeutics

Active, not recruiting

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

France, United Kingdom, New Zealand, Sweden
Alnylam Pharmaceuticals

Approved for marketing

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
Ionis Pharmaceuticals, Inc.
AstraZeneca

Active, not recruiting

The CARDIO-TTRansform Scintigraphy Sub-study

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

United States, Spain
Alnylam Pharmaceuticals

Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
Alnylam Pharmaceuticals

Active, not recruiting

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
Alnylam Pharmaceuticals

Active, not recruiting

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more
Ionis Pharmaceuticals, Inc.
AstraZeneca

Active, not recruiting

CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

United States, United Kingdom, Italy, Spain
Intellia Therapeutics
Regeneron Pharmaceuticals

Recruiting

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, United Kingdom, New Zealand, Canada
Pfizer

Recruiting

Vyndaqel Capsules Special Investigation (ATTR-CM)

Transthyretin (TTR) Amyloid Cardiomyopathy

Japan
Zhejiang University

Recruiting

Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy

Transthyroxin Amyloidosis Cardiomyopathy

China
Thibaud Damy

Completed

Prevalence of Transthyretin Amyloidosis in Hypertrophic Cardiomyopathy (Amylo)

Cardiac Amyloidosis | Hypertrophic Cardiomyopathy (HCM) | Amyloidosis in Transthyretin (TTR)

France
Steen Hvitfeldt Poulsen
Novo Nordisk A/S

Recruiting

Phenotyping and Characterization of wtATTR-CM (TRACE 1)

Quality of Life | Transthyretin Amyloidosis | Transthyretin Amyloid Cardiomyopathy | Wild-Type Transthyretin-Related (ATTR)Amyloidosis

Denmark
Pfizer

Not yet recruiting

Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

United States
Alnylam Pharmaceuticals

Completed

A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)

United Kingdom
Alnylam Pharmaceuticals

Recruiting

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) (ConTTRibute)

Transthyretin-Mediated Amyloidosis | ATTR Amyloidosis

Israel, Italy, Netherlands, Spain, United States, France, Denmark, Germany, Portugal, Brazil, Taiwan
Alnylam Pharmaceuticals

Completed

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

TTR-mediated Amyloidosis

Portugal, Spain, Sweden, Brazil, United States, France, Germany
Alnylam Pharmaceuticals

Completed

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

TTR-mediated Amyloidosis

United Kingdom
Alnylam Pharmaceuticals

Completed

Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects

TTR-mediated Amyloidosis

United Kingdom
Pfizer

Completed

This Study Will Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis

Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype

United States
Ionis Pharmaceuticals, Inc.

Completed

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Ionis Pharmaceuticals, Inc.

Completed

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

New Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Alnylam Pharmaceuticals

Completed

Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

TTR-mediated Amyloidosis

United States, United Kingdom
Pfizer

Completed

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy (ATTR-ACT)

Transthyretin (TTR) Amyloid Cardiomyopathy

United States, Spain, Czechia, Canada, Belgium, Netherlands, Germany, Japan, France, United Kingdom, Brazil, Italy, Sweden
Alnylam Pharmaceuticals

Completed

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)

TTR-mediated Amyloidosis

Portugal, Sweden, Brazil, United States, France, Spain, Germany
Alnylam Pharmaceuticals

Completed

A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

TTR-mediated Amyloidosis

United States, Canada, United Kingdom
Steen Hvitfeldt Poulsen

Recruiting

Prevalence of wtATTR-CM After Carpal Tunnel Release Surgery (CACTuS2)

Transthyretin Amyloidosis | Transthyretin Cardiac Amyloidosis | Wild-Type Transthyretin-Related (ATTR)Amyloidosis

Denmark
Pfizer

Completed

Prevalence and Characteristics of Transthyretin Amyloidosis in Patients With Left Ventricular Hypertrophy of Unknown Etiology (TTRACK)

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)

Italy, Romania, France, Australia, Austria, Portugal, Slovakia, Slovenia, Spain, United Kingdom
Alnylam Pharmaceuticals

Recruiting

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects

Transthyretin-Mediated Amyloidosis

United Kingdom
Pfizer

Completed

Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

Transthyretin (TTR) Amyloid Cardiomyopathy

United States, Taiwan, Canada, Belgium, Australia, Czechia, Sweden, Spain, Italy, Hong Kong, Germany, Netherlands, United Kingdom, France, Japan, Argentina, Brazil
Pfizer

Recruiting

Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid Cardiomyopathy

ATTR-CM (Transthyretin Amyloid Cardiomyopathy)

Korea, Republic of
Novo Nordisk A/S

Active, not recruiting

A Research Study to Look at How a New Medicine Called NNC6019-0001 Works and How Safe it is for People Who Have Heart Disease Due to Transthyretin (TTR) Amyloidosis

Transthyretin Amyloid Cardiomyopathy (ATTR CM)

United States, Netherlands, Spain, Portugal, Canada, Czechia, France, Germany, Italy, Japan
Austin Neuromuscular Center
Alnylam Pharmaceuticals

Recruiting

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

Polyneuropathies | Transthyretin Amyloidosis | Wild-Type Transthyretin-Related (ATTR)Amyloidosis | Wild-Type Transthyretin Cardiac Amyloidosis | Wild Type ATTR Amyloidosis

United States
Mahidol University
Pfizer

Not yet recruiting

Thailand ATTR-CM Registry

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Thailand
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP) (BioTRAP)

Transthyretin Amyloidosis | Transthyretin Amyloid Cardiopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

India, Germany
Eidos Therapeutics, a BridgeBio company

Completed

Study of AG10 in Amyloid Cardiomyopathy

Familial ATTR-CM (ATTRm-CM, or FAC) | Wild-type ATTR-CM (ATTRwt-CM)

United States
Algalarrondo Vincent
Bichat Hospital; Bioquantis

Recruiting

Artificial Intelligence to Assist the Echocardiographic Identification of Transthyretin Cardiac Amyloidosis (AI-ATTR-ECHO)

Transthyretin Cardiac Amyloidosis | Transthyretin Amyloid Cardiomyopathy | Amyloid Cardiomyopathy

France
Novo Nordisk A/S

Recruiting

A Research Study to Look at Long-term Treatment With a Medicine Called NNC6019-0001 for People Who Have Heart Failure Due to Transthyretin Amyloidosis

Transthyretin Amyloid Cardiomyopathy (ATTR CM)

United States, Netherlands, Germany, Canada, Czechia, France, Italy, Portugal, Spain
AstraZeneca

Recruiting

Transthyretin Amyloidosis Cardiomyopathy in Patients With HFpEF in Russia (TETRAMER)

Transthyretin Amyloidosis Cardiomyopathy, Heart Failure With Preserved Ejection Fraction

Russian Federation

Clinical Trials on Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

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