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NCT Number: NCT03362164

Evaluation of HEArt invoLvement 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.

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Key information

Conditions

Rare Diseases Aortic Stenosis, Subvalvular Aortic Valve Disease Aortic Valve Stenosis Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiomyopathies Cardiomyopathy, Hypertrophic Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Chronic Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Fabry Disease Fabry Disease, Cardiac Variant Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Fibrosis Genetic Diseases, Inborn Genetic Diseases, X-Linked Heart Diseases Heart Valve Diseases Hypertrophic Cardiomyopathy Kidney Diseases Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Renal Insufficiency Renal Insufficiency, Chronic Sphingolipidoses Stroke Urogenital Diseases Urologic Diseases Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Wuerzburg University Hospital

Würzburg, Bavaria, 97080, Germany

Location status: Recruiting

Location contact

Irina Schumacher

CONTACT

[email protected]

004993120139714

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • No informed consent
  • Withdrawal of informed consent

Treatment and study plan

Primary outcomes

  1. Cardiac death

    Time frame: From date of inclusion until the date of first documented event, up to the year 2032

    Patients sustaining cardiac death

Secondary outcomes

  1. 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

  2. Malign Arrhythmias

    Time frame: From date of inclusion until the date of death, up to the year 2032

    Patients suffering any malign arrhythmias

Study contacts

Contact information is provided by the study sponsor or research team.

Jonas Muentze, MD

CONTACT

[email protected]

004993120139958

Peter Nordbeck, MD, PhD

CONTACT

[email protected]

004993120139181

Sponsors and collaborators

Lead sponsor

Wuerzburg University Hospital

Other

Collaborators

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

Registry information

Official study title

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

Acronym: HEAL-FABRY

Important dates

Study start
2001
Primary completion
2032
Study completion
2032
First posted
Dec 5, 2017
Registry last updated
Jul 7, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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