Clinical Trials on Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)

Total 80 results

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)
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
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
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
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
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

No longer available

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
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
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
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
Eidos Therapeutics, a BridgeBio company

Withdrawn

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

United States
Pfizer

Completed

The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin

Transthyretin Familial Amyloid Polyneuropathy

Japan
Pfizer

Completed

The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

Transthyretin Amyloid Polyneuropathy (ATTR-PN)

China
Pfizer

Completed

Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)

Transthyretin Familial Amyloid Poluneuropathy
Alnylam Pharmaceuticals

Completed

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

Transthyretin (TTR)-Mediated Amyloidosis

United Kingdom
Pfizer

Completed

Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv) Patients Included in B3461028 and B3461045. (TRAMA)

Polyneuropathy | Hereditary Transthyretin Amyloidosis (ATTRv)

Spain
Akcea Therapeutics
United BioSource, LLC

Recruiting

A Non-interventional Cohort Safety Study of Patients With hATTR-PN

Hereditary Transthyretin Amyloidosis With Polyneuropthy

United States, France, Greece, Italy, Spain, Canada, Germany, Portugal
Akcea Therapeutics

Recruiting

A Clinical Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Patients With hATTR-PN

Hereditary Transthyretin Amyloidosis With Polyneuropthy

United States, Canada
Pfizer

Completed

The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis

Transthyretin-associated Amyloidosis With Polyneuropathy

United States, France, Germany, Italy
SOM Innovation Biotech SA
Hospital Vall d'Hebron

Completed

Study of SOM0226 in Familial Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy (FAP)

Spain
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
Peking University Third Hospital

Recruiting

The Effect of Tafamidis on Transthyretin Stabilization, Safety, Tolerability and Efficacy in Transthyretin Amyloid Polyneuropathy Patients

Transthyretin Amyloidosis

China
AstraZeneca

Recruiting

hATTR Polyneuropathy in Russia (PRIMER)

Hereditary Transthyretin Amyloidosis

Russian Federation
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
Rennes University Hospital

Recruiting

Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran (MyocardON-TTR)

Transthyretin Amyloidosis | Amyloidosis, Hereditary

France
Pfizer

Completed

An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy | ATTR-PN

Sweden, Brazil, Germany, Argentina, France, Portugal
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
IRCCS Policlinico S. Matteo

Recruiting

The Italian Transthyretin Amyloidosis Web-Network (ITA-WebNet)

ATTR Amyloidosis

Italy
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
Prothena Biosciences Limited

Terminated

A Study of PRX004 in Subjects With Amyloid Transthyretin (ATTR) Amyloidosis

Amyloidogenic Transthyretin (ATTR) Amyloidosis

United States, Spain, Sweden, Portugal
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
Alnylam Pharmaceuticals

Not yet recruiting

Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy

Hereditary Amyloidosis, Transthyretin-Related | Asymptomatic Carrier State

France
Umeå University

Completed

The Effect of Diflunisal on Familial Transthyretin Amyloidosis (DFNS01)

Amyloidosis

Sweden
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
Boston University
National Institute of Neurological Disorders and Stroke (NINDS); Food and Drug...

Completed

The Effect of Diflunisal on Familial Amyloidosis

Familial Amyloid Polyneuropathy | Familial Amyloidosis

United States, Italy, Japan, Sweden, 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
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Exploring Biomarkers in Hereditary Transthyretin Amyloidosis (ELBA)

Hereditary Transthyretin Amyloidosis

Italy
University of Edinburgh
British Heart Foundation; Netherlands Heart Foundation; Deutsches Zentrum für...

Recruiting

Quantitative-imaging in Cardiac Transthyretin Amyloidosis (I-CARE)

ATTR-CM

Netherlands
Akcea Therapeutics

Recruiting

TEGSEDI Pregnancy Surveillance Program

Pregnancy | Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy

United States
Pfizer

Completed

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

Familial Amyloid Polyneuropathy

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

Recruiting

A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

United States, Italy, Portugal, Spain, Taiwan, Sweden, Canada, Brazil, France, New Zealand, Argentina, Cyprus, Australia, Turkey
Ionis Pharmaceuticals, Inc.

Completed

NEURO-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 Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

United States, Spain, Taiwan, Italy, Germany, Argentina, Australia, Brazil, Canada, Cyprus, France, Greece, New Zealand, Portugal, Sweden, Turkey
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

Completed

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

Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)

United Kingdom
Pfizer

Recruiting

Vyndaqel Capsules Special Investigation (ATTR-CM)

Transthyretin (TTR) Amyloid Cardiomyopathy

Japan
Thibaud Damy

Completed

Prevalence of Transthyretin Amyloidosis in Hypertrophic Cardiomyopathy (Amylo)

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

France

Clinical Trials on Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP)

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