Clinical Trials on Transthyretin (TTR)-Mediated Amyloidosis

Total 144 results

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

Completed

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

Transthyretin Mediated Amyloidosis (ATTR)

United Kingdom, France, Portugal, Sweden
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

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

Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)

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
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
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
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

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

TTR-mediated Amyloidosis

United States, United Kingdom
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
Alnylam Pharmaceuticals

Recruiting

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

Transthyretin-Mediated Amyloidosis

United Kingdom
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
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
IRCCS Policlinico S. Matteo

Recruiting

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

ATTR Amyloidosis

Italy
Prothena Biosciences Limited

Terminated

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

Amyloidogenic Transthyretin (ATTR) Amyloidosis

United States, Spain, Sweden, Portugal
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
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)
Akcea Therapeutics

Recruiting

TEGSEDI Pregnancy Surveillance Program

Pregnancy | Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy

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

Recruiting

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

ATTR-CM

Netherlands
Zhejiang University

Recruiting

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

Transthyroxin Amyloidosis Cardiomyopathy

China
Ionis Pharmaceuticals, Inc.
AstraZeneca

Active, not recruiting

The CARDIO-TTRansform Scintigraphy Sub-study

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

United States, Spain
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
Boston University
Corino Therapeutics, Inc.

Completed

Short-term Effects of TOLCAPONE on Transthyretin Stability in Subjects With Leptomeningeal TTR Amyloidosis (ATTR)

Transthyretin Amyloidosis | Amyloidosis, Leptomeningeal, Transthyretin-Related

United States
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
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
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
Alnylam Pharmaceuticals

Recruiting

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

Polyneuropathy | Hereditary Transthyretin-mediated (hATTR) Amyloidosis

Netherlands, Spain, United States, Germany, France, Italy, Portugal
Mayo Clinic

Completed

Long-term Monitoring of Patients With Cardiac Amyloidosis With Implantable Event Monitors

TTR Cardiac Amyloidosis

United States
Alnylam Pharmaceuticals

Completed

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Polyneuropathy | Hereditary Transthyretin-mediated (ATTRv) Amyloidosis

United States
Pfizer

Completed

The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

Transthyretin Amyloid Polyneuropathy (ATTR-PN)

China
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
Oncology Institute of Southern Switzerland
University of Kiel; Ente Ospedaliero Cantonale, Bellinzona

Completed

Prevalence of Wild Type ATTR

Wild-type Transthyretin Amyloidosis

Switzerland
IRCCS Policlinico S. Matteo

Completed

A Study of Doxycycline and Tauroursodeoxycholic Acid (Doxy/TUDCA) Plus Standard Supportive Therapy Versus Standard Supportive Therapy Alone in Cardiac Amyloidosis Caused by Transthyretin

TTR Cardiac Amyloidosis

Italy
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
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
Pr. Nicolas GIRERD

Recruiting

Prognostic Value of Echocardiographic Parameters Based on Machine Learning Approach (ATTR-AI)

Transthyretin Cardiac Amyloidosis

France
Poitiers University Hospital
Pfizer

Completed

Imaging for the Evaluation of Transthyretin Cardiac Amyloidosis Prevalence of Patients Over 65 Hospitalized in Cardiology Wards (the IMPACT Study) (IMPACT)

Transthyretin Cardiac Amyloidosis

France
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
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
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
Columbia University
Attralus, Inc.

Recruiting

Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR

Transthyretin Amyloidosis

United States

Clinical Trials on Transthyretin (TTR)-Mediated Amyloidosis

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