Active clinical trials for "Amyloidosis"

Florbetapir F 18 is an experimental radioactive drug that may allow doctors to image changes in the brain using a PET (Positron Emission Tomography) scanner. The purpose of this study is to evaluate the imaging characteristics of, Florbetapir F 18 (also known as 18F-AV-45) in patients who have previously undergone bleeding in their brains. Florbetapir F 18 binds to amyloid-ß peptide (Aß) that accumulates in the brains of patients with bleeding. These accumulations are called amyloid plaques and when extensive are labeled cerebral amyloid angiopathy (CAA). Florbetapir F 18 sticks to the amyloid plaques in the brain and emits a low level of gamma rays which can be detected by a PET camera. MRI detected microbleeds have been identified as markers of clinically silent hemorrhage from bleeding-prone vessels. Another imaging marker of vessel damage and risk of bleeding is the spot sign (SS). Finally, certain genetic signatures (ApoE genotype) have been shown to be associated with Aß deposition in the brain or predispose patients to higher risks of bleeding. This research study will explore the interactions of these factors and understand the physiology of intracerebral bleeding.

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 Polyneuropathy from blood and number of correctly identified patients with Transthyretin-Related Familial Amyloidotic Polyneuropathy

Those with abnormal vital signs after TAVR need to be willing to obtain a bone scan to evaluate for wildtype amyloidosis. Positive bone scan findings will require evaluation for primary amyloidosis with blood and urine monoclonal immunoglobulin testing. Primary amyloidosis is a different type of disease which requires different treatment.

Cardiac amyloidosis describes a process by which abnormally folded proteins infiltrate the heart tissue. Given the insidious nature of this disease process, diagnosis is often too late for a meaningful intervention. Advances in the treatment of the amyloidoses have improved outcomes for patients with these conditions. The focus of this study is to identify the involvement of the heart, most closely associated with mortality, so that aggressive management can be instituted improving prognosis.

This is a cross sectional study to estimate the prevalence of the presence of amyloid deposits in a biopsy of subcutaneous fat cell, carpal flexor retinaculum and synovial tissue sheath of the flexor tendons requirement for carpal tunnel surgery.

This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to Venetoclax prior to approval by the local regulatory agency. Availability will depend on territory eligibility. A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.

The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).

The purpose of this study is to Early identify patients based on clinical manifestation, imaging, gene and histology, explore diagnostic tools get gene repertoire of Chinese Build a cerebral amyloid angiopathy (CAA) prospective cohorts, observing the disease history, and exploring prognostic factors of hemorrhage in CAA patients

Aortic stenosis (AS) is the most common valvular heart disease. Once symptomatic with severe AS, outcome is poor unless the valve is replaced surgically or via transcatheter aortic valve replacement (TAVR). Transthyretin amyloid (ATTR) deposits are common in the heart muscle in up to 25% of octogenarians, and after an asymptomatic period of unknown duration, cause overt heart failure and arrhythmias in a proportion of cases. The prevalence and impact of covert ATTR amyloidosis in elderly individuals with AS are unknown. Detection would avoid misdiagnosis, guide treatment and, potentially, improve outcomes. Recent data have shown that echocardiography, cardiovascular magnetic resonance (CMR), computed tomography (CT), and DPD scintigraphy, can identify ATTR amyloid deposits, but the clinical performance of these various tests is unknown. This study will investigate elderly patients with symptomatic severe AS using imaging to explore ATTR amyloid in AS and determine its prevalence and impact on outcome. The investigators aim to recruit a total of 250 patients aged 75 or older being considered for intervention for severe AS. The prevalence of cardiac amyloid will be assessed in three arms (sAVR, TAVI and medical therapy, with a likely patient ratio of 50:150:50), using five investigation modalities - all cohorts (echocardiography and DPD scintigraphy); sAVR cohort (biopsy and CMR); TAVI cohort (EqCT); medical therapy only cohort (as per work-up/trial prior to no intervention decision). The primary outcome measure is patient mortality. Secondary outcomes measures are major adverse cardiovascular events, length of stay, pacemaker implantation, ECV measured by EqCT and CMR. Follow up will be at 1-year with clinical echocardiogram (for sAVR and TAVI patients) and/or telephone interview for all patients (if not carried out in person at the time of the echocardiogram).