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:
Use samples procured from patients to improve understanding of molecular, cellular, and tissue-level processes produced by a variety of cardiac diseases and therapeutic interventions.
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.