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

July 6, 2022 updated by: PD Dr. Peter Nordbeck, Wuerzburg University Hospital

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

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

Study Overview

Status

Recruiting

Conditions

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 Type

Observational

Enrollment (Anticipated)

650

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Peter Nordbeck, MD, PhD
  • Phone Number: 004993120139181
  • Email: nordbeck_p@ukw.de

Study Contact Backup

Study Locations

  • Germany
    • Bayern
      • Würzburg, Bayern, Germany, 97080
        • Recruiting
        • Wuerzburg University Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The study population includes every patient with Fabry disease who is seen in the FAZiT Wuerzburg. No limitations are made.

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • No informed consent
  • Withdrawal of informed consent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Heart transplantation
Time Frame: 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
From date of inclusion until the date of first documented event, up to the year 2032
Malign Arrhythmias
Time Frame: From date of inclusion until the date of death, up to the year 2032
Patients suffering any malign arrhythmias
From date of inclusion until the date of death, up to the year 2032

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Wuerzburg University Hospital

Collaborators

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

Investigators

  • Principal Investigator: Peter Nordbeck, MD, PhD, Wuerzburg University Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2001

Primary Completion (Anticipated)

March 1, 2032

Study Completion (Anticipated)

March 1, 2032

Study Registration Dates

First Submitted

November 29, 2017

First Submitted That Met QC Criteria

November 29, 2017

First Posted (Actual)

December 5, 2017

Study Record Updates

Last Update Posted (Actual)

July 7, 2022

Last Update Submitted That Met QC Criteria

July 6, 2022

Last Verified

July 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FAZiT-2001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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