Clinical Research Directory

Korean Post-marketing Surveillance Vyndamax® Capsules for the Treatment of Transthyretin Amyloid Cardiomyopathy

This non-interventioal study will be performed by design of post-marketing surveillance (PMS) as an additional pharmacovigilance activity of the Risk Management Plan (RMP) for Vyndamax® Capsules, which is required by the Ministry of Food and Drug Safety (MFDS) according to the local regulation. This post-marketing surveillance will investigate the safety and effectiveness of Vyndamax® Capsules as the treatment of transthyretin amyloid cardiomyopathy during 10 years under the setting of routine practice in Korea.

A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany

This observational study aims to describe the characteristics, health outcomes, and epidemiology of individuals diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Germany. ATTR-CM is a rare and serious heart disease caused by the buildup of an abnormal protein called transthyretin in the heart muscle. This can lead to heart failure and other cardiovascular complications. This is a retrospective, non-interventional study based on anonymized statutory health insurance claims data from Germany. The study includes adults with a new diagnosis of ATTR-CM identified between 2020 and 2022. Data from both inpatient and outpatient healthcare settings are analyzed. No participants are recruited, and no medical treatments, procedures, or study visits are assigned as part of this study. The study will describe overall survival, cardiovascular-related hospitalizations, and the incidence of ATTR-CM in Germany. A predefined subgroup of individuals with at least one documented prescription for tafamidis will be analyzed separately to describe outcomes in this population. In addition, the study will examine the use of genetic testing after diagnosis and describe patient characteristics, as observed in routine clinical care. By using nationwide healthcare data, this study aims to provide a comprehensive and recent real-world overview of ATTR-CM in Germany and to support a better understanding of the disease.

US Benchmarking Clinical Study

The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin amyloidosis with cardiomyopathy (ATTR-CM), and in control subjects without heart failure or cardiomyopathy. The main question it aims to answer is: • Can SWV be used to measure myocardial and liver stiffness in the study's target populations? Researchers will compare patients with ATTR-CM, patients without ATTR-CM, and a control group to determine the distributions of SWV in each population. Participants will: * Have a standard cardiology assessment, including a physical exam, blood work, and an echocardiogram. * Undergo an investigational assessment with the eMyosound LYRA device to measure SWV in their myocardium and liver. * Have the investigational assessment repeated by a second observer to assess measurement reliability.