Clinical Research Directory

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

The purpose of this study is to: * Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events * Evaluate the efficacy of nucresiran compared to placebo on additional assessments of CV events and/or death * Evaluate the efficacy of nucresiran compared to placebo on patient-reported health status and health-related quality of life

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

The purpose of this study is to: * Describe the clinical characteristics of adult patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) treated with vutrisiran in routine clinical care * Describe treatment patterns of adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Assess health-related quality of life (HRQOL) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Assess healthcare resource use (HCRU) in adult patients with ATTR-CM treated with vutrisiran in routine clinical care * Compare the long-term effectiveness of vutrisiran versus other approved ATTR-CM treatments in routine clinical care

Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations

The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of eplontersen; all are areas of missing information Primary objectives are: 1. To describe demographic and clinical characteristics of patients at eplontersen initiation, including the prevalence of prior liver transplant (overall and by reason for liver transplant), and the prevalence of severe hepatic impairment; and to describe patients in these subgroups (prior liver transplant, severe hepatic impairment). 2. To describe long-term safety in patients who initiate eplontersen treatment, including onset of new clinical events, abnormal laboratory values and serious adverse events.