NCT03362164

Evaluation of HEArt invoLvement in Patients With FABRY Disease

Prospective Monocentric Cohort Study to Evaluate Predictors for Heart Failure and Sudden Cardiac Death in Patients With Fabry Disease

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 650 (estimated)
• Sponsor: Wuerzburg University Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: —

Summary

This study evaluates predictors for the incidence of arrhythmias and sudden cardiac death as well as terminal heart failure in patients with Fabry disease.

Detailed description

Fabry disease is a rare disease and part of the group of lysosomal storage disorders. Natural history of Fabry disease has proven poor survival to ages \>50 years outlining the importance to evaluate cardiac symptoms and outcomes of patients with Fabry disease. This study is a prospective cohort study and observes patients since 2001. Through this long-term experience and the relative high number of patients this study is suggested to help estimating the risk of cardiac arrhythmias and sudden cardiac death (SCD) as well as death or heart transplantation due to terminal heart failure. All patients in treatment in the Fabry Center Wuerzburg (FAZiT) are included in this study if informed consent is provided.

Conditions

• Rare Diseases
• Fabry Disease
• Fabry Disease, Cardiac Variant
• Hypertrophic Cardiomyopathy

Timeline

• Start date: 2001-01-01
• Primary completion: 2032-03-01
• Completion: 2032-03-01
• First posted: 2017-12-05
• Last updated: 2022-07-07

Locations

1 site across 1 country: Germany