Clinical trials: Familial Amyloid Polyneuropathies
Amyloid Cardiomyopathy, Transthyretin-Related
Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10
This is a single center, prospective, randomized, placebo-controlled study of AG10 in healthy adult subjects
- Trial name
- NCT03294707
- Testing
- AG10 oral tablet
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Amyloidosis; Systemic, Amyloidosis Cardiac
Essen Amyloidosis Registry
The Essen Amyloidosis Registry (EAR) is a prospective, observational registry designed to collect comprehensive clinical data on patients diagnosed with systemic amyloidosis.
- Trial name
- EAR (NCT06887283)
- Where
- Germany
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- University Hospital, Essen
Amyloid Cardiomyopathy, Wild Type ATTR Amyloidosis
Association Between Lifetime Physical Activity and Exercise and the Development of Wild-type Transthyretin Amyloid Cardiomyopathy
The aim of this study is to investigate the association between increased lifetime physical activity and the development of wild-type transthyretin amyloid cardiomyopathy.
- Trial name
- NCT06261216
- Testing
- Interview
- Where
- Austria
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Danon Disease, Fabry Disease
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
What is this study about? This research is focused on improving the care for people with rare heart muscle diseases, known as rare cardiomyopathies.
- Conditions
- Danon Disease, Fabry Disease, Cardiac Amyloidosis, Noonan Syndrome
- Trial name
- NCT07336394
- Where
- China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Chinese Academy of Medical Sciences, Fuwai Hospital
Cardiac Amyloidosis
Imaging Cardiac Amyloidosis: A Pilot Study Using F-18 Florbetapir Positron Emission Tomography
The primary aim of this pilot study is to determine whether amyloid deposits in the heart can be measured non-invasively by F-18 florbetapir (Trade Name: Amyvid) positron emission tomography (PET) in 30 individuals with…
- Trial name
- NCT01683825
- Testing
- F-18 florbetapir PET
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
Amyloid, Cardiac Amyloidosis
Pan-Amyloid PET/CT in Various Amyloid-Related Disease
To evaluate the potential usefulness of 18F-92/AV45/TPZA/FT8, 11C-PIB positron emission tomography/computed tomography (PET/CT) for the diagnosis of primary and metastatic lesions in various Pan-Amyloid-related disease patients.
- Conditions
- Amyloid, Cardiac Amyloidosis, Amyloidosis
- Trial name
- NCT07130565
- Testing
- 18F-92/AV45/TPZA/FT8, 11CPIB
- Where
- 3 sites in China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Tianjin Medical University
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
- Trial name
- NCT02797847
- Testing
- ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis Cardiac
Ultrasound Therapy In Cardiac Amyloidosis
This is a prospective pilot clinical study of subjects with cardiac amyloidosis and control subjects without amyloidosis where we plan to evaluate changes in myocardial blood flow, systolic and diastolic function before and after…
- Trial name
- UTICA (NCT04667494)
- Testing
- Sonotherapy
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS
Amyloidosis, Cardiac Amyloidosis
Study of Olfactory Disorders in Patients With Cardiac Amyloidosis
Amyloidosis is a disease caused by the continuous accumulation of fibrillary proteins in the extracellular matrix causing the architecture of different organs to be disrupted. The prevalence of the disease increases with age.
- Conditions
- Amyloidosis, Cardiac Amyloidosis, Olfactory Loss, Hyposmia
- Trial name
- AMYLODOR (NCT04661800)
- Testing
- Sniffin's stick test
- Where
- France
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Amyloidosis, AL Amyloidosis
BE.Amycon Biobank & Data Registry UZ Leuven
The goal of this study is to collect and store human body material (HBM) of patients with amyloidosis in a biobank "BE.Amycon biobank" for future research and to collect clinical data of patients with amyloidosis in a database…
- Conditions
- Amyloidosis, AL Amyloidosis, Amyloidosis Cardiac
- Trial name
- NCT07172243
- Where
- 2 sites in Belgium
- Volunteers
- 505 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Wild-type ATTR Amyloidosis, Amyloidosis Cardiac
ATTR Amyloid Cardiomyopathy: Characterization of Extracellular Vesicles as Potential Disease Stratifiers and Prognostic Biomarkers
This study explores whether extracellular vesicles (EVs) tiny particles released into the bloodstream by cells can serve as early and minimally invasive biomarkers for transthyretin amyloid cardiomyopathy (ATTR-CM).
- Conditions
- Wild-type ATTR Amyloidosis, Amyloidosis Cardiac, Amyloidosis, Hereditary, Amyloidosis Transthyretin
- Trial name
- EV-ATTR (NCT07314268)
- Where
- Brazil
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo General Hospital
Amyloidosis Cardiac, Virtual Reality
Virtual Reality Assisted Patient Empowerment: Diagnose ATTR-Amyloidosis And Start Treatment
ATTR amyloidosis is a serious condition with significant morbidity and mortality. In Germany, there are numerous unreported cases of untreated patients, and diagnosing and initiating treatment often requires multiple specialized…
- Trial name
- VRAP-DAAST (NCT05938218)
- Testing
- Intervention
- Where
- Germany
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Heinrich-Heine University, Duesseldorf
Multiple Myeloma, Amyloidosis Cardiac
The Diagnostic Pattern and Prognosis of Multiple Myeloma Patients With Myocardial Amyloidosis Were Evaluated by NMR Based Metabolomics
In this clinical study, a single-center retrospective cohort study was used to explore the clinical characteristics and risk factors of patients with multiple myeloma myocardial amyloidosis.
- Trial name
- NCT05718401
- Testing
- NMR
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital Xi'an Jiaotong University
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
Hereditary Amyloidosis, Transthyretin-Related
Early Detection of Neuropathy in ATTRv
The purpose of the study is to evaluate and compare different tools that are used to detect evidence of peripheral neuropathy in patients with TTRv.
- Trial name
- EDONA (NCT05311488)
- Testing
- neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Amyloid Neuropathies, Transthyretin Amyloidosis
Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study
Familial amyloid neuropathies (FAP) are hereditary disease due to a mutation of the tranthyretin gene (TTR). These neuropathies are severe and life frightening.
- Trial name
- NCT03588468
- Testing
- Electrophysiological biomarkers, MRI biomarkers
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
Cardiac Amyloidosis
Molecular Imaging of Myocardial Fibrosis in Cardiac Amyloidosis
The primary aim of our pilot study is to determine whether fibrosis in the heart can be measured with [68Ga]CBP8, a positron emission tomography (PET) probe, using PET/magnetic resonance imaging (MRI) imaging, in 30 individuals…
- Trial name
- NCT05619302
- Testing
- [68Ga]CBP8 PET/MRI
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
Amyloid, Cardiac Amyloidosis
[18F]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis
[18F]FT8, a derivative of 1-(4-pyridyl)-4-piperazinyl arene ([18F]TPZA), exhibits high affinity for AL amyloid in myocardial tissue sections and shows no significant binding to transthyretin amyloid.
- Conditions
- Amyloid, Cardiac Amyloidosis, Amyloidosis, Healthy Subjects
- Trial name
- NCT07232459
- Testing
- [18F]FT8
- Where
- 3 sites in China
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Tianjin Medical University