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

Sponsors

Lead Sponsor: Wuerzburg University Hospital

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

Source 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.

Overall Status Recruiting
Start Date January 2001
Completion Date March 2032
Primary Completion Date March 2032
Study Type Observational
Primary Outcome
Measure Time Frame
Cardiac death From date of inclusion until the date of first documented event, up to the year 2032
Secondary Outcome
Measure Time Frame
Heart transplantation From date of inclusion until the date of first documented event, up to the year 2032
Malign Arrhythmias From date of inclusion until the date of death, up to the year 2032
Enrollment 650
Condition
Eligibility

Sampling Method: Non-Probability Sample

Criteria:

Inclusion Criteria:

- Fabry disease (genetically confirmed)

- Signed informed consent

- 18 years and older

Exclusion Criteria:

- No informed consent

- Withdrawal of informed consent

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Overall Official
Last Name Role Affiliation
Peter Nordbeck, MD, PhD Principal Investigator Wuerzburg University Hospital
Overall Contact

Last Name: Peter Nordbeck, MD, PhD

Phone: 004993120139181

Email: [email protected]

Location
Facility: Status: Contact: Wuerzburg University Hospital Irina Schumacher 004993120139714 [email protected]
Location Countries

Germany

Verification Date

February 2020

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Wuerzburg University Hospital

Investigator Full Name: PD Dr. Peter Nordbeck

Investigator Title: Head Consultant Cardiology and Chief of Electrophysiology

Keywords
Has Expanded Access No
Condition Browse
Acronym HEAL-FABRY
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

Source: ClinicalTrials.gov