Clinical trials: Hereditary Transthyretin Amyloidosis
ATTR-CM
Implementation of Standardized Early Identification and Diagnosis for Transthyretin Amyloidosis (ATTR) in High-Risk Populations
This multicenter, prospective, single-arm, interventional study will enroll about 4,000 adult patients at high risk of ATTR amyloidosis from around 50 sites in China.
- Trial name
- NCT07338942
- Testing
- implementation of standard early identification and diagnosis of ATTR
- Where
- 48 sites in China
- Volunteers
- 4,000 people
- Sponsor
- AstraZeneca
Cardiovascular Diseases, Hypertrophic Cardiomyopathy (HCM)
AI-enabled Screening and Diagnosis of Cardiomyopathies Using Coronary CTA
The goal of this observational and diagnostic study is to develop and validate an artificial intelligence assisted approach for coronary computer tomography angiography-(CCTA)-based screening and diagnosis of cardiomyopathies in…
- Conditions
- Cardiovascular Diseases, Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Restrictive Cardiomyopathy
- Trial name
- Atlantis (NCT06748261)
- Testing
- CCTAI model
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Shanghai Zhongshan Hospital
Polyneuropathies, Wild Type ATTR Amyloidosis
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
To evaluate the efficacy and safety of patisiran in patients with wtATTR amyloidosis and symptomatic polyneuropathy by evaluating the effect on neurologic impairment and quality of life.
- Conditions
- Polyneuropathies, Wild Type ATTR Amyloidosis, Wild-Type Transthyretin-Related (ATTR)Amyloidosis, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT05023889
- Testing
- patisiran
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Austin Neuromuscular Center
Lumbar Spinal Stenosis, Cardiac Amyloidosis
Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis
Primary objective: To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective.
- Conditions
- Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation
- Trial name
- NCT06034405
- Testing
- Tc99-PYP or Tc99-HDMP Scan
- Where
- 6 sites in United States
- Volunteers
- 1,663 people, healthy volunteers welcome
- Sponsor
- Columbia University
Cardiac Amyloidosis
Thalidomide/Dexamethasone Treatment And PET Evaluation In Organ Involvemenet of Cardiac Amyloidosis
To prove the organ-reversing potential of thalidomide for amyloidosis with cardiac involvement
- Trial name
- TaPiOCA (NCT02966522)
- Testing
- Thalidomide, Dexamethasone
- Where
- South Korea
- Volunteers
- 30 people
- Sponsor
- Seoul National University Hospital
Amyloidosis, Hereditary, Transthyretin-Related
Exercise in Hereditary ATTR (ATTRv) Amyloidosis
This study aims to explore the effects of a 12-week exercise intervention on the physical and mental health of people living with Hereditary ATTR (ATTRv) Amyloidosis, a rare genetic disease caused by mutations in the…
- Trial name
- NCT07033715
- Testing
- Exercise
- Where
- Portugal
- Volunteers
- 42 people
- Sponsor
- University of Maia
Amyloidosis Cardiac
Multimodality Cardiac Imaging for Disease Progression in ATTR-CM
The goal of this clinical trial is to investigate whether new imaging techniques can help us to better understand the cardiac amyloidosis.
- Trial name
- FAPI-ATTR (NCT07112066)
- Testing
- FAPI tracer
- Where
- Switzerland
- Volunteers
- 50 people
- Sponsor
- Dominik Benz
Familial Amyloid Polyneuropathy, Familial Amyloidosis
The Effect of Diflunisal on Familial Amyloidosis
The purpose of this study is to determine if diflunisal can prevent progressive lower leg nerve damage in patients with familial amyloidosis polyneuropathy.
- Trial name
- NCT00294671
- Testing
- diflunisal
- Where
- 8 sites in United States, Italy and 3 more
- Volunteers
- 130 people
- Sponsor
- Boston University
Cardiac Amyloidosis
Morpho-functional Cardiac Modifications in Treated Mutated Transthyretin Cardiac Amyloidosis
Cardiac Amyloidosis (CA) is characterized by a long subclinical phase characterized by deposition of amyloid fibrils in atria, valves and walls of ventricles.
- Trial name
- NCT04387344
- Where
- 2 sites in Italy
- Volunteers
- 25 people
- Sponsor
- University of Messina
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle.
- Trial name
- ACO-REAL (NCT07235462)
- Testing
- Acoramidis (356 mg film-coated tablets)
- Where
- 17 sites in Austria, Belgium and 15 more
- Volunteers
- 2,000 people
- Sponsor
- Bayer
Amyloidosis Cardiac, Carpal Tunnel Syndrome
Prevalence of Wild Type Transthyretin Cardiac Amyloidosis in Patients Operated for Idiopathic Carpal Tunnel Syndrome
Wild-type transthyretin cardiac amyloidosis is an underdiagnosed depository disease in which fibril monomers of misfolded amyloid protein accumulates in various tissues, including the heart, and cause tissue dysfunction.
- Trial name
- CACTuS (NCT03996382)
- Testing
- DPD Scintigraphy
- Where
- Denmark
- Volunteers
- 120 people
- Sponsor
- Aarhus University Hospital Skejby
Cardiac Amyloidosis
PET/MR Imaging In Patients With Cardiac Amyloidosis
PET scanning (positron emission tomography) is a well-established technique used to identify areas of interest within the body. It involves injecting a radioactive tracer which highlights abnormal areas.
- Trial name
- NCT03626584
- Testing
- 18F-NaF PET
- Where
- United Kingdom
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- University of Edinburgh
Cardiac Amyloidosis
Mitochondrial Function in Transthyretin Amyloidosis
Hereditary (familial) amyloidosis arising from the misfolding of a mutated or variant transthyretin, is the most frequent form of amyloid cardiomyopathy in the Caribbean basin.
- Trial name
- MIT-Amylose (NCT03328338)
- Where
- Martinique
- Volunteers
- 12 people
- Sponsor
- University Hospital Center of Martinique
Amyloidosis, Transthyretin Amyloidosis
Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis
This is a single center prospective study evaluating 124I-evuzumitide in patients with systemic amyloidosis. The purpose of this study is to 1) Establish the diagnostic accuracy of 124I-evuzumitide in cardiac amyloidosis 2)…
- Trial name
- NCT05758493
- Testing
- 124I-Evuzamitide
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Oregon Health and Science University
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Amyloidosis, Amyloid Cardiomyopathy
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- ATTRibute-CM (NCT03860935)
- Testing
- acoramidis
- Where
- 104 sites in United States, Australia and 16 more
- Volunteers
- 632 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Cardiac Amyloidosis
Screening Algorithm for Early Detection of Occult Cardiac Amyloidosis
Early diagnosis of cardiac amyloidosis (CA) is crucial because of the poor overall survival, high mortality and the need for early therapy including new treatment possibilities for transthyretin amyloidosis.
- Trial name
- EARLY-CA (NCT05693376)
- Where
- Germany
- Volunteers
- 85 people, healthy volunteers welcome
- Sponsor
- University Hospital, Essen
Cardiac Amyloidosis
MECKI-Amyloidosis: Assessment of the Exercise Capacity and Prognosis of Patients with Cardiac Amyloidosis
This study aims to advance the understanding of cardiac amyloidosis, its effects on exercise capacity, and its prognostic implications.
- Trial name
- MECKI-AMI (NCT06799273)
- Where
- Italy
- Volunteers
- 1,400 people
- Sponsor
- Centro Cardiologico Monzino
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Cardiac Amyloidosis
PET/MRI Evaluation of Cardiac Amyloid
Cardiac amyloidosis is a disorder characterized by the deposition of abnormal proteins called amyloid in the heart tissue. This makes it difficult for the heart to function properly.
- Trial name
- NCT03119558
- Testing
- 18F-Florbetaben (Neuraceq®) PET/MRI
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Stanford University