Evaluation of HEArt invoLvement in Patients With FABRY Disease (HEAL-FABRY)
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|ClinicalTrials.gov Identifier: NCT03362164|
Recruitment Status : Recruiting
First Posted : December 5, 2017
Last Update Posted : February 10, 2020
|Condition or disease|
|Rare Diseases Fabry Disease Fabry Disease, Cardiac Variant Hypertrophic Cardiomyopathy|
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|
|Estimated Enrollment :||650 participants|
|Official Title:||Prospective Monocentric Cohort Study to Evaluate Predictors for Heart Failure and Sudden Cardiac Death in Patients With Fabry Disease|
|Actual Study Start Date :||January 2001|
|Estimated Primary Completion Date :||March 2032|
|Estimated Study Completion Date :||March 2032|
- Cardiac death [ Time Frame: From date of inclusion until the date of first documented event, up to the year 2032 ]Patients sustaining cardiac death
- 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
- Malign Arrhythmias [ Time Frame: From date of inclusion until the date of death, up to the year 2032 ]Patients suffering any malign arrhythmias
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03362164
|Contact: Peter Nordbeck, MD, PhDemail@example.com|
|Contact: Jonas Muentze, MDfirstname.lastname@example.org|
|Wuerzburg University Hospital||Recruiting|
|Würzburg, Bayern, Germany, 97080|
|Contact: Irina Schumacher 004993120139714 email@example.com|
|Principal Investigator:||Peter Nordbeck, MD, PhD||Wuerzburg University Hospital|