Clinical trials: ATTR
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)
Phase 3 efficacy and safety of acoramidis in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- NCT04882735
- Testing
- Acoramidis
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Transthyretin Amyloid Cardiomyopathy ("ATTR-CM")
A Prospective Study to Assess the Utility of CA-4F in a Canadian Cardiology Setting
This study will assess whether CA-4F can improve diagnosis of ATTR-CM in a community cardiology setting when used to screen electronic medical record data for patients suitable for follow-up investigations.
- Trial name
- NCT05210010
- Testing
- CA-4F
- Where
- 3 sites in Canada
- Volunteers
- 8 people
- Sponsor
- Ensho Health Intelligent Systems Inc.
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Single-arm, Multi-center Study Evaluating the Acceptance and Feasibility of a Digital Remote Monitoring Tool Configured for Patients With ATTR-CM: ACO-Monitor
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a heart condition caused by a protein called transthyretin (TTR) building up as amyloid in the heart muscle.
- Trial name
- ACO-MONITOR (NCT07557147)
- Testing
- Luscii
- Where
- 3 sites in Austria, Germany and 1 more
- Volunteers
- 60 people
- Sponsor
- Bayer
Transthyretin Amyloid Cardiomyopathy
A Study to Assess the Safety and Efficacy Of Tafamidis In Chinese Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
This is a national, multi-center, single-arm study, open-label to patients with symptomatic Transthyretin amyloid cardiomyopathy (ATTR-CM) who are tafamidis naïve.
- Trial name
- NCT04814186
- Testing
- Tafamidis
- Where
- 9 sites in China
- Volunteers
- 53 people
- Sponsor
- Pfizer
Transthyretin Amyloid Cardiomyopathy (ATTR CM)
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
This study will test a medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis. It will look at how safe this medicine is in the long term and if it can reduce symptoms of a heart disease due to TTR…
- Trial name
- NCT06260709
- Testing
- NNC6019-0001
- Where
- 38 sites in United States, Canada and 8 more
- Volunteers
- 88 people
- Sponsor
- Novo Nordisk A/S
Transthyretin Amyloid Cardiomyopathy
A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis
Transthyretin (TTR) is a protein made by the liver that helps transport thyroid hormone and vitamin A in the blood. In some people, this protein breaks down and forms harmful clumps called amyloid.
- Trial name
- ACO-SWITCH (NCT07298044)
- Testing
- Acoramidis
- Where
- 26 sites in Austria, Belgium and 3 more
- Volunteers
- 50 people
- Sponsor
- Bayer
TTR-mediated Amyloidosis
A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.
- Trial name
- NCT02292186
- Testing
- Revusiran (ALN-TTRSC)
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia
This is a multicenter observational study consisting of retrospective and prospective phases. The retrospective phase will entail secondary data collection from electronic or paper medical records of patients who underwent…
- Conditions
- Hereditary Transthyretin Amyloidosis, Carpal Tunnel Syndrome, Polyneuropathy, Amyloidosis
- Trial name
- LOCUS (NCT06414746)
- Where
- 13 sites in Russia
- Volunteers
- 721 people
- Sponsor
- AstraZeneca
Familial Amyloid Polyneuropathy, ATTR-PN
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.
- Trial name
- NCT00791492
- Testing
- Fx-1006A
- Where
- 11 sites in Argentina, Brazil and 4 more
- Volunteers
- 86 people
- Sponsor
- Pfizer
Transthyretin Amyloid Cardiomyopathy
Retinal Fundus Imaging and OCT Imaging for Ocular Detection of ATTR-CM
This multicenter pilot study will enroll adult participants with a confirmed clinical diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) and heart failure (HF) controls with documentation within the past 2 years that…
- Trial name
- NCT07690436
- Where
- 20 sites in United States, Germany and 3 more
- Volunteers
- 500 people
- Sponsor
- AstraZeneca
Transthyretin (TTR) Amyloid Cardiomyopathy
Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy
- Trial name
- NCT02791230
- Testing
- Tafamidis
- Where
- 80 sites in United States, Argentina and 15 more
- Volunteers
- 1,733 people
- Sponsor
- Pfizer
Transthyretin-type Cardiac Amyloidosis
Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
- Trial name
- ATTR-CM (NCT07306949)
- Testing
- Acoramidis
- Where
- 16 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hereditary Transthyretin Amyloidosis (ATTRv) Cardiomyopathy (CM), Mixed Phenotype
A Study to Examine the Clinical Effectiveness of Tafamidis in Patients With Mixed Phenotype Hereditary Transthyretin Amyloidosis
This study will examine the clinical effectiveness of Tafamidis in patients with Mixed Phenotype Hereditary Transthyretin Amyloidosis using data that already exist in patients' medical records.
- Trial name
- NCT05139680
- Testing
- tafamidis
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Pfizer
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study
The main purpose of this study is to measure the amyloid burden, defined as extracellular volume (ECV) assessed by Magnetic Resonance Imaging (MRI) over time in a subset of up to 150 participants enrolled in ION-682884-CS2…
- Trial name
- NCT06073574
- Testing
- Cardiac MRI
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 160 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy
Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations
The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of…
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
- Trial name
- sTTRing (NCT07654855)
- Testing
- None ( observational study )
- Where
- 6 sites in United States, Canada and 4 more
- Volunteers
- 320 people
- Sponsor
- AstraZeneca
Neuromuscular Disease, Neuromuscular Diseases (NMD)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary
- Trial name
- NCT06672237
- Testing
- nexiguran ziclumeran
- Where
- 14 sites in Argentina, Brazil and 4 more
- Volunteers
- 60 people
- Sponsor
- Intellia Therapeutics
Amyloid Cardiomyopathy, Transthyretin-Related
Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
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)
- Trial name
- AG10 (NCT04988386)
- Testing
- Acoramidis (AG10)
- Where
- 72 sites in United States, Australia and 15 more
- Volunteers
- 389 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Polyneuropathy, Amyloid, Neuropathic Pain
Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP)
An International, multicenter, epidemiological observational study investigating the prevalence of Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR-FAP) in participants with small fiber polyneuropathy of no obvious…
- Conditions
- Polyneuropathy, Amyloid, Neuropathic Pain, Cardiac Failure, Orthostatic Hypotension
- Trial name
- TRAP2-1 (NCT01705626)
- Where
- 11 sites in Austria, Hungary and 4 more
- Volunteers
- 500 people
- Sponsor
- CENTOGENE GmbH Rostock
ATTR-CM
A Retrospective Validation Study To Identify Chart-Based Clinical Diagnosis Of Wild-Type Transthyretin Amyloid Cardiomyopathy (Attrwt-CM) And Non-Amyloid Heart Failure Among Patients With Heart Failure (HF).
This is an observational, retrospective non-inferiority study with a study sample from a large national database. A machine learning (ML) model will use a national database to predict the clinical diagnosis of ATTRwt-CM among HF…
- Trial name
- NCT06029452
- Testing
- Machine learning algorithm
- Where
- United States
- Volunteers
- 558 people
- Sponsor
- Pfizer
Healthy Participants
A First-In-Human Study of the Study Medicine, Called PF-07291177, in Healthy Adult Participants
The purpose of the study is to learn about the safety, tolerability (the extent to which side effects can be tolerated), and plasma pharmacokinetics (PK) (PK helps us understand how the drug is changed and eliminated from body…
- Trial name
- NCT05434091
- Testing
- PF-07291177
- Volunteers
- healthy volunteers welcome
- Sponsor
- Pfizer