Organ manifestations and long-term outcome of Fabry disease in patients with the GLA haplotype D313Y.
Daniel Oder, Nurcan Üçeyler, Dan Liu...
https://pubmed.ncbi.nlm.nih.gov/27059467Actively Recruiting
Age: 18Years +
All Genders
ID03362164
Prospective Cohort Study to Evaluate Heart Failure and Sudden Cardiac Death Risks in Patients With Fabry Disease
Led by Wuerzburg University Hospital · Updated on 2022-07-07
650
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
W
Wuerzburg University Hospital
Lead Sponsor
C
Competence Network Heart Failure
Collaborating Sponsor
AI-Summary
What this Trial Is About
Fabry disease is a rare lysosomal storage disorder with a natural history that leads to poor survival beyond age 50, highlighting the need to understand cardiac symptoms and outcomes in these patients. Researchers are studying predictors for heart rhythm problems, sudden cardiac death, and terminal heart failure in people with Fabry disease. This is a long-term, prospective cohort study that has been observing patients since 2001 to estimate these risks. The study includes all patients treated at the Fabry Center Wuerzburg who provide informed consent. It follows patients over time to monitor occurrences of cardiac death, heart transplants, and dangerous heart rhythm disturbances. The observation period extends until the year 2032 or until the first event of interest occurs for each participant. Participants will be regularly evaluated for heart-related events and outcomes throughout the study period. Researchers will track the time from enrollment to cardiac death, heart transplantation, or malignant arrhythmias. This ongoing monitoring aims to improve understanding of heart involvement in Fabry disease and guide future care. Total participation time varies based on individual timing of events or study end in 2032.
CONDITIONS
Brief Title
Evaluation of HEArt invoLvement in Patients With FABRY Disease
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Genetically confirmed Fabry disease
- Signed informed consent
- 18 years and older
You will not qualify if you...
- No informed consent
- Withdrawal of informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Long-term Monitoring
Duration - Up to 31 years
Participants with genetically confirmed Fabry disease are observed over time to evaluate heart failure and sudden cardiac death risks.
Regular visits as part of routine care at the Fabry Center Wuerzburg
Trial Site Locations
Total: 1 location
1
Wuerzburg University Hospital
Würzburg, Bavaria, Germany, 97080
Actively Recruiting
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Research Team
P
Peter Nordbeck, MD, PhD
J
Jonas Muentze, MD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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Published Research Related To This Trial
High-Sensitivity Troponin: A Clinical Blood Biomarker for Staging Cardiomyopathy in Fabry Disease.
Nora Seydelmann, Dan Liu, Johannes Krämer...
https://pubmed.ncbi.nlm.nih.gov/27247331Usefulness of an Implantable Loop Recorder to Detect Clinically Relevant Arrhythmias in Patients With Advanced Fabry Cardiomyopathy.
Frank Weidemann, Sebastian K G Maier, Stefan Störk...
https://pubmed.ncbi.nlm.nih.gov/27265676Impact of immunosuppressive therapy on therapy-neutralizing antibodies in transplanted patients with Fabry disease.
M Lenders, D Oder, A Nowak...
https://pubmed.ncbi.nlm.nih.gov/28682471α-Galactosidase A Genotype N215S Induces a Specific Cardiac Variant of Fabry Disease.
Daniel Oder, Dan Liu, Kai Hu...
https://pubmed.ncbi.nlm.nih.gov/29018006Cardiac and renal dysfunction is associated with progressive hearing loss in patients with Fabry disease.
Maria Köping, Wafaa Shehata-Dieler, Mario Cebulla...
https://pubmed.ncbi.nlm.nih.gov/29161295Intraepidermal nerve fiber density as a predictor of cardiac events in Fabry disease.
Kolja Lau, Dan Liu, Victoria Sokalski...
https://pubmed.ncbi.nlm.nih.gov/42004908Gene variants of unknown significance in Fabry disease: Clinical characteristics of c.376A>G (p.Ser126Gly).
Kolja Lau, Nurcan Üçeyler, Tereza Cairns...
https://pubmed.ncbi.nlm.nih.gov/35212486Association and diagnostic utility of diastolic dysfunction and myocardial fibrosis in patients with Fabry disease.
Dan Liu, Daniel Oder, Tim Salinger...
https://pubmed.ncbi.nlm.nih.gov/30018776Value of the CHA2DS2-VASc score and Fabry-specific score for predicting new-onset or recurrent stroke/TIA in Fabry disease patients without atrial fibrillation.
Dan Liu, Kai Hu, Marie Schmidt...
https://pubmed.ncbi.nlm.nih.gov/29797054