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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03362164

Acronym

HEAL-FABRY

Enrollment

650

Registered

2017-12-05

Start date

2001-01-31

Completion date

2032-03-31

Last updated

2022-07-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Keywords

Fabry, Fibrosis, Dialysis, Stroke, Chronic kidney disease

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.

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Inclusion criteria

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

Exclusion criteria

* No informed consent * Withdrawal of informed consent

Design outcomes

Primary

Measure	Time frame	Description
Cardiac death	From date of inclusion until the date of first documented event, up to the year 2032	Patients sustaining cardiac death

Secondary

Measure	Time frame	Description
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

Countries

Germany

Contacts

Primary ContactPeter Nordbeck, MD, PhD

nordbeck_p@ukw.de004993120139181

Backup ContactJonas Muentze, MD

muentze_j@ukw.de004993120139958

Outcome results