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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 : December 5, 2017
Sponsor:
Collaborators:
Competence Network Heart Failure
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
St George's, University of London
Information provided by (Responsible Party):
PD Dr. Peter Nordbeck, Wuerzburg University Hospital

November 29, 2017
December 5, 2017
December 5, 2017
January 2001
March 2032   (Final data collection date for primary outcome measure)
Cardiac death [ Time Frame: From date of inclusion until the date of first documented event, up to the year 2032 ]
Patients sustaining cardiac death
Same as current
No Changes Posted
  • 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
Same as current
Not Provided
Not Provided
 
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
This study evaluates predictors for the incidence of arrhythmias and sudden cardiac death as well as terminal heart failure in patients with Fabry disease.

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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples With DNA
Description:
ECG, Holter ECG, Echo, Cardiac MRI, Endomyocardial Biopsy, Kidney Biopsy, Cranial MRI, Skin Biopsy, EDTA Blood, Serum, Plasma, Urine
Non-Probability Sample
The study population includes every patient with Fabry disease who is seen in the FAZiT Wuerzburg. No limitations are made.
  • Rare Diseases
  • Fabry Disease
  • Fabry Disease, Cardiac Variant
  • Hypertrophic Cardiomyopathy
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
650
Same as current
March 2032
March 2032   (Final data collection date for primary outcome measure)

Inclusion Criteria:

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

Exclusion Criteria:

  • No informed consent
  • Withdrawal of informed consent
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Not Provided
Contact: Peter Nordbeck, MD, PhD 004993120139181 nordbeck_p@ukw.de
Contact: Jonas Muentze, MD 004993120139958 muentze_j@ukw.de
Germany
 
 
NCT03362164
FAZiT-2001
No
Not Provided
Not Provided
PD Dr. Peter Nordbeck, Wuerzburg University Hospital
Wuerzburg University Hospital
  • Competence Network Heart Failure
  • Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
  • St George's, University of London
Principal Investigator: Peter Nordbeck, MD, PhD Wuerzburg University Hospital
Wuerzburg University Hospital
November 2017