Evaluation of HEArt invoLvement in Patients With FABRY Disease

Fabry Disease Clinical Trial

— HEAL-FABRY  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03362164
• Other study ID #: FAZiT-2001
• Status: Recruiting
• Start date: January 2001
• Est. completion date: March 2032

Study information

• Verified date: July 2022
• Source: Wuerzburg University Hospital
• Contact: Peter Nordbeck, MD, PhD
• Phone: 004993120139181
• Email: nordbeck_p[@]ukw.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 650
• Est. completion date: March 2032
• Est. primary completion date: March 2032
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Fabry disease (genetically confirmed)

• Signed informed consent

• 18 years and older

Exclusion Criteria:

• No informed consent

• Withdrawal of informed consent

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Hypertrophic
• Fabry Disease
• Fabry Disease, Cardiac Variant
• Hypertrophic Cardiomyopathy
• Rare Diseases

Locations

Country	Name	City	State
Germany	Wuerzburg University Hospital	Würzburg	Bayern

Sponsors (4)

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

Country where clinical trial is conducted

Germany, 

References & Publications (6)

Köping M, Shehata-Dieler W, Cebulla M, Rak K, Oder D, Müntze J, Nordbeck P, Wanner C, Hagen R, Schraven S. Cardiac and renal dysfunction is associated with progressive hearing loss in patients with Fabry disease. PLoS One. 2017 Nov 21;12(11):e0188103. doi — View Citation

Lenders M, Oder D, Nowak A, Canaan-Kühl S, Arash-Kaps L, Drechsler C, Schmitz B, Nordbeck P, Hennermann JB, Kampmann C, Reuter S, Brand SM, Wanner C, Brand E. Impact of immunosuppressive therapy on therapy-neutralizing antibodies in transplanted patients — View Citation

Oder D, Liu D, Hu K, Üçeyler N, Salinger T, Müntze J, Lorenz K, Kandolf R, Gröne HJ, Sommer C, Ertl G, Wanner C, Nordbeck P. a-Galactosidase A Genotype N215S Induces a Specific Cardiac Variant of Fabry Disease. Circ Cardiovasc Genet. 2017 Oct;10(5). pii: — View Citation

Oder D, Üçeyler N, Liu D, Hu K, Petritsch B, Sommer C, Ertl G, Wanner C, Nordbeck P. Organ manifestations and long-term outcome of Fabry disease in patients with the GLA haplotype D313Y. BMJ Open. 2016 Apr 8;6(4):e010422. doi: 10.1136/bmjopen-2015-010422. — View Citation

Seydelmann N, Liu D, Krämer J, Drechsler C, Hu K, Nordbeck P, Schneider A, Störk S, Bijnens B, Ertl G, Wanner C, Weidemann F. High-Sensitivity Troponin: A Clinical Blood Biomarker for Staging Cardiomyopathy in Fabry Disease. J Am Heart Assoc. 2016 May 31; — View Citation

Weidemann F, Maier SK, Störk S, Brunner T, Liu D, Hu K, Seydelmann N, Schneider A, Becher J, Canan-Kühl S, Blaschke D, Bijnens B, Ertl G, Wanner C, Nordbeck P. Usefulness of an Implantable Loop Recorder to Detect Clinically Relevant Arrhythmias in Patient — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cardiac death	Patients sustaining cardiac death	From date of inclusion until the date of first documented event, up to the year 2032
Secondary	Heart transplantation	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
Secondary	Malign Arrhythmias	Patients suffering any malign arrhythmias	From date of inclusion until the date of death, up to the year 2032