Evaluation of HEArt invoLvement in Patients With FABRY Disease

Recruiting

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

• Registration Number: NCT03362164
• Lead Sponsor: Wuerzburg University Hospital

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.

Study Timeline

• Study Start: 2001-01
• Study First Submitted: 2017-11-29
• Study First Submitted QC: 2017-11-29
• Study First Posted: 2017-12-05
• Status Verified: 2022-07
• Last Update Submitted: 2022-07-06
• Last Update Posted: 2022-07-07
• Primary Completion: 2032-03
• Study Completion: 2032-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 650

Inclusion Criteria

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

Exclusion Criteria

• No informed consent
• Withdrawal of informed consent

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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

Trial Locations

Locations (1)

Wuerzburg University Hospital; 🇩🇪 Würzburg, Bayern, Germany