Clinical trials: Familial Amyloid Polyneuropathies
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels
The goal of this study is to learn whether PET-CT imaging using evuzamitide can help diagnose transthyretin cardiac amyloidosis (ATTR-CA) in patients whose standard nuclear imaging results are unclear but who have elevated TAD1…
- Trial name
- TRACE (NCT07538518)
- Testing
- Evuzamitide
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Lorena Saelices
Cardiac Amyloidosis
Cardiac Amyloidosis Registry of University Hospital Leipzig
This is a clinical registry of patients with cardiac amyloidosis being treated at University Hospital Leipzig. The aim of the registry is to collect detailed information about clinical events, symptoms, imaging, biomarkers…
- Trial name
- NCT06129656
- Testing
- Routine diagnostics, Routine treatment
- Where
- Germany
- Volunteers
- 500 people
- Sponsor
- University of Leipzig
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Aortic Stenosis Symptomatic, Amyloidosis Cardiac
ATTR Amyloidosis in Elderly Patients With Aortic Stenosis
Severe aortic stenosis is defined with a mean transvalvular pressure gradient (MTPG) > 40mmHg and a calculated aortic valve area of < 1cm2.
- Trial name
- NCT04061213
- Testing
- Tc-99m-DPD scintigraphy
- Where
- Switzerland
- Volunteers
- 489 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
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
Cardiomyopathies, Amyloidosis Cardiac
Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
The study aims to identify new diagnostic and prognostic markers for CMP that can help predict disease progression. In particular, the study will focus on microRNAs (miRNAs) and spatial transcriptomics, which are emerging…
- Conditions
- Cardiomyopathies, Amyloidosis Cardiac, Fabry Disease, Arrhythmogenic Cardiomyopathy
- Trial name
- CMPMIRNA (NCT06813443)
- Where
- 3 sites in Italy
- Volunteers
- 700 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals
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
Hereditary Transthyretin Amyloidosis
hATTR Polyneuropathy in Russia
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There…
- Trial name
- PRIMER (NCT06365593)
- Where
- 3 sites in Russia
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
AL Amyloidosis (AL), Cardiac Amyloidosis
Multimodal Radiomics Model (18F-FAPI PET/CT + CMR) for AL Cardiac Amyloidosis Prognosis
1. Goal of the Study: The goal of this prospective observational study is to develop and validate a novel, non-invasive method for predicting the prognosis of patients with light-chain cardiac amyloidosis (AL-CA).
- Trial name
- AL-CA (NCT07103863)
- Where
- China
- Volunteers
- 49 people
- Sponsor
- Beijing Anzhen Hospital
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
Registre HEAR, Healthcare European Amyloidosis Registry
This is a non-interventional, prospective, retrospective, non-comparative, multi-center study. In order not to interfere with patient management, the study is observational. Thus, no follow-up visit is imposed.
- Trial name
- NCT05101304
- Where
- France
- Volunteers
- 5,000 people
- Sponsor
- Saving Lives Matters
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Transthyretin Familial Amyloid Polyneuropathy
Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions [ADRs]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received…
- Trial name
- NCT02146378
- Testing
- Vyndaqel
- Volunteers
- 500 people
- Sponsor
- Pfizer
Cardiac Amyloidosis
Validation of Different Diagnostic Modalities in the Detection of Cardiac Amyloidosis Among Patients With "Red Flags"
The study aims to test and compare the diagnostic yield of different diagnostic modalities (speckle tracking echocardiography, CMR, serum and urine protein analysis and bone tracer scintigraphy) in the detection of cardiac…
- Trial name
- NCT06175858
- Testing
- CMR
- Volunteers
- 30 people
- Sponsor
- Assiut University
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