HEAL-FABRY: Evaluation of HEArt invoLvement in Patients With FABRY Disease

Sponsor

Wuerzburg University Hospital (Other)

Overall Status

Recruiting

CT.gov ID

NCT03362164

Collaborator

Competence Network Heart Failure (Other), Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) (Other), St George's, University of London (Other)

650

Enrollment

Location

373.9

Anticipated Duration (Months)

1.7

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Rare Diseases Fabry Disease Fabry Disease, Cardiac Variant Hypertrophic Cardiomyopathy

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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

650 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

Jan 1, 2001

Anticipated Primary Completion Date :

Mar 1, 2032

Anticipated Study Completion Date :

Mar 1, 2032

Outcome Measures

Primary Outcome Measures

Cardiac death [From date of inclusion until the date of first documented event, up to the year 2032]
Patients sustaining cardiac death

Secondary Outcome Measures

Heart transplantation [From date of inclusion until the date of first documented event, up to the year 2032]
On the basis of severe cardiac damage heart transplantation is needed
Malign Arrhythmias [From date of inclusion until the date of death, up to the year 2032]
Patients suffering any malign arrhythmias