Evaluation of HEArt invoLvement in Patients With FABRY Disease (HEAL-FABRY)

Wuerzburg University Hospital

Status: Enrolling

Conditions: Fabry Disease; Rare Diseases; Hypertrophic Cardiomyopathy; Fabry Disease, Cardiac Variant

Study type: Observational

Funder types: Other

Identifiers

NCT03362164

FAZiT-2001

Details and patient eligibility

About

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

Full 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.

Enrollment: 650 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Fabry disease (genetically confirmed)
• Signed informed consent
• 18 years and older

Exclusion criteria

• No informed consent
• Withdrawal of informed consent