The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events.
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.
The specific aims of this epidemiologic investigation include:
1. To identify subjects with previous lumbar spinal stenosis (LSS) Surgery who have evidence of transthyretin (TTR) amyloid deposits in spinal specimens and could be at risk for ATTR cardiac amyloidosis.
2. To evaluate for ATTR-CA among those with localized TTR in the spinal tissue.
Exercise training in patients with heart failure and preserved ejection fraction (HFpEF) has been associated with an improvement in cardiorespiratory fitness and quality of life.
The primary purpose of this protocol is to create a registry of patients with plasma cell disorders (PCDs), including for example the cancer multiple myeloma (MM), who complete the assessment, previously known as a "geriatric assessment," as is outlined in this protocol. Secondary objectives include measuring the response rate to participation of patients in this study, assessing patient satisfaction with the questionnaire, and gathering information that would lend support for future research into these types of assessments in patients with PCDs.
This is a Phase 2 open-label extension study to evaluate the long-term safety, tolerability, and clinical activity of AT-02.
AT-02 is an investigational medicinal product being developed to treat systemic amyloidosis.
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
This is a single center prospective study evaluating 124I-evuzumitide in patients with systemic amyloidosis. The purpose of this study is to 1)identify and characterize the distribution and uptake of 124I-evuzumitide in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and 2) Correlate the uptake with the structure and function of different organs, including the heart. To achieve these goals, eligible patients will undergo primarily hybrid positron emission tomography and magnetic resonance imaging (PET/MRI).
RATIONALE: Collecting and storing samples of blood, urine, tissue and data from patients with amyloid diseases to test in the laboratory may help the study of this disease in the future.
PURPOSE: This research study is collecting samples from patients with amyloid diseases
The goal of this clinical trial is to learn about malnutrition and weight loss in patients with Amyloidosis.
The main question it aims to answer is:
Is it feasible to use a low-cost nutrition-based application (apps) for use on a smartphone to obtain detailed information on caloric intake in Amyloidosis patients
Participants will be asked to:
This phase I/II trial tests the safety, side effects, and best dose of venetoclax, daratumumab, and dexamethasone for the treatment of systemic light-chain amyloidosis in patients with a deoxyribonucleic acid (DNA) abnormality called a translocation involving chromosomes 11 and 14, or "t(11;14)". Venetoclax works by attaching to a protein called Bcl-2, in order to kill cancer cells. Daratumumab works by binding to a target on the surface of cancer cells called Cluster of differentiation 38 (CD38).