Active clinical trials for "Amyloidosis"

To determine whether protective antibody levels increase after booster dosing with the Moderna COVID-19 vaccine in patients diagnosed with Hematologic Malignancies who have low antibody levels after a prior first vaccination with any of the SARS-CoV2 vaccines that were authorized for use in the USA. Researchers will also assess whether the booster dosing with the Moderna COVID-19 vaccine is safe in patients with multiple myeloma, amyloidosis, or other blood cancers.

AL amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract. The primary purpose of this study is to determine the recommended dose of CAEL-101 to facilitate progression of further clinical trials and evaluate safety and tolerability of CAEL-101 in combination with the standard of care (SoC) cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy and daratumumab .

Heart failure is defined as the inability of the heart to provide sufficient output to meet the needs of the body. It can occur in the course of a myocardial infarction, angina pectoris, hypertension, etc. Its frequency increases with age. It is a major public health problem. Heart failure first appears during exercise, then at rest. Initially, the heart tries to adapt to the loss of its contraction force by accelerating its beats (increase in heart rate), then it increases in volume (thickening of the walls or dilation of the cardiac cavities). This extra workload for the heart eventually leads to heart failure. Cardiac amyloidosis is a possible cause of the disease in the West Indian population. Cardiac amyloidosis is a rare disease related to our own proteins that will accumulate and cluster together to form abnormal protein deposits that will eventually lead to heart failure. Cardiac amyloidosis particularly affects West Indians, due to the high frequency in this population of a genetic anomaly associated with the disease: the Valine 122 Isoleucine (Val122l) mutation of the transthyretin gene (protein transthyretin in which isoleucine is substituted for valine at position 122 (Ile 122)). Early detection of amyloidosis appears essential for the implementation of appropriate therapies and therefore for an improvement in patient survival. For this it seems important to better specify the frequency of cardiac amyloidosis in heart failure in the French West Indies.

Recently, treatment with tafamidis in patients with cardiac ATTR lead to a significant reduction in mortality. The Perugini score is commonly used on planar bone scans to differentiate cardiac ATTR from other amyloidosis or normal patients but fails to evaluate amyloid burden and patient prognosis. Although semi-quantitative methods have been suggested to evaluate the amyloid burden, there a need for quantitative methods for longitudinal assessment of the disease.

The purpose of this study is to evaluate the prevalence of amyloidosis in population of patient affected by suspect bilateral carpal tunnel with indication of surgery

Introduction: Transthyretin cardiac amyloidosis (ATTR) is an important cause of heart failure. Cardiac planar radionuclide imaging using 99mTc-labeled bone seeking radiopharmaceuticals is used as a noninvasive diagnostic criterion in patients without detectable monoclonal protein. The visual assessment remains the main noninvasive criterion for the diagnosis. Medical therapy using tafamidis meglumine that binds to transthyretin and prevents amyloidogenesis, recently demonstrated a reduction in all-cause mortality and cardiovascular-related hospitalizations. As a consequence, there is a need for quantitative approaches that would be useful for diagnosis and prognosis assessment but also for the evaluation of patient therapeutic response. Materials and methods: The investigators aim to include 35 patients with a suspected diagnosis of cardiac ATTR amyloidosis in whom a cardiac planar radionuclide imaging using 99mTc-labeled bone seeking radiopharmaceuticals is planned as part of routine noninvasive diagnosis work-up. Using a test-retest approach, the aim is to compare a quantitative method vs. conventional semi-quantitative approaches for the assessment of cardiac uptake of bone radiopharmaceuticals using new 3D CZT-based SPECT-CT cameras in patients with suspected cardiac ATTR amyloidosis. The investigators estimated that 20 patients will have a diagnosis of cardiac ATTR amyloidosis. In the latter patients, the aim is to evaluate the impact of 6-month therapy using tafamidis on quantitative and semi-quantitative assessment of cardiac uptake of bone radiopharmaceuticals Perspectives: This new non invasive imaging techniques for the quantitative assessment of the amyloid burden in patients with cardiac ATTR amyloidosis may help identify the responders and the patients who should benefit from dose intensification.

This will be a systematic, combined, prospective assessment of the novel echographic, CMR, and PET imaging tools in newly-diagnosed patients with cardiac AL amyloidosis at baseline and after treatment.

To investigate to what extent chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) is part of early non-cardiac manifestations of wild-type TTR cardiac amyloidosis (wtTTR-CA). Consequently, explore whether this could ultimately lead to faster diagnosis and clinical outcome of wild-type TTR cardiac amyloidosis (wtTTR-CA).

Although being classified as a rare disease, cardiac amyloidosis constitutes an increasing cause of heart failure, which is often overlooked and thus poorly managed. Amyloidosis involves deposits of light chain immunoglobulins in the immunoglobulin light chain amyloidosis (AL) type, but it may also be of a hereditary type in mutated transthyretin amyloidosis (ATTRm) or of a senile type in wildtype transthyretin forms (ATTRwt). Myocardial biopsy remains a gold standard for definitive diagnosis but it is a traumatic technique which only provides information on a limited number of sampled sites. Useful but not fully specific signs of cardiac amyloidosis may also be provided by Magnetic Resonance Imaging or MRI (delayed retention imaging) and echocardiography (longitudinal strain pattern). Notwithstanding the above, relatively specific markers of amyloid plaques are now available in Positron Emission Tomography (PET). These markers are primarily fluorinated tracers which have been developed for the diagnosis of Alzheimer's disease. Two of these have already been the subject of feasibility studies in the setting of cardiac amyloidosis diagnosis, on a maximum of 10 amyloidosis patients but with very favorable results. The hypothesis is that one of these two tracers, Florbetaben labelled with Fluorine-18-Florbetaben (18F-Florbetaben) used in the study, has sufficiently strong and prolonged binding kinetics at the level of the amyloid plaques to allow: (i) achieving whole-body PET recordings and thus, (ii) identifying not only cardiac amyloidosis but also extracardiac binding sites, particularly those readily accessible to biopsy sampling. This hypothesis has been strengthened by a recent case report illustrating the ability of whole-body florbetaben-PET to image not only cardiac but also extra-cardiac sites of amyloid deposits (Clin Nucl Med. 2017;42(1):50-3).

Cardiac amyloidosis is a restrictive cardiomyopathy with a potentially severe prognosis that can be life-threatening. It is linked in the vast majority of cases to a light chain deposition of immunoglobulin or transthyretin. Although myocardial involvement is predominant, other locations are possible: the atrioventricular conduction system, coronary arteries and valve leaflets. In systematic histological analyzes, deposits of amyloidosis infiltrating the aortic valve have been reported with a frequency of up to 74% for degenerative RA. The nature of these deposits has never been established because the immunostaining carried out all remained negative, probably due to decalcification prior to cutting. Currently, these deposits are considered to be local degenerative phenomena without clinical repercussions. However, the use of bone scintigraphy has shown a high prevalence, between 14 and 16%, of ATTR cardiac amyloidosis in patients with severe RA. The diagnosis of ATTR amyloidosis has been proven histologically in a few patients. Sequencing of the TTR gene has shown that they are mainly wild forms. In fact, the prevalence of transthyretin mutations in our local cohort is 20%. The objective of this study is to determine by proteomic analysis based on mass spectrometry, the prevalence of ATTR aortic valve amyloidosis in patients undergoing surgical valve replacement for degenerative aortic stenosis.