Evaluating 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 looks at patients with Fabry disease to see what factors might lead to heart problems like arrhythmias and heart failure.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 650 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Wuerzburg University Hospital Academic / other |
| Locations | 1 site (Würzburg, Bavaria) |
| Trial ID | NCT03362164 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on patients with Fabry disease to evaluate predictors for the incidence of arrhythmias, sudden cardiac death, and terminal heart failure. It is a prospective cohort study that has been observing patients since 2001, leveraging long-term data to estimate cardiac risks associated with this rare lysosomal storage disorder. The study includes all patients receiving treatment at the Fabry Center Wuerzburg who provide informed consent.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with genetically confirmed Fabry disease.
Not a fit: Patients who do not provide informed consent or withdraw consent will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could improve understanding of cardiac risks in Fabry disease patients, potentially leading to better management and treatment strategies.
How similar studies have performed: While the study addresses a rare condition, similar observational studies have shown success in understanding cardiac implications in other lysosomal storage disorders.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Fabry disease (genetically confirmed) * Signed informed consent * 18 years and older Exclusion Criteria: * No informed consent * Withdrawal of informed consent
Where this trial is running
Würzburg, Bavaria
- Wuerzburg University Hospital — Würzburg, Bavaria, Germany (Recruiting)
Study contacts
- Principal investigator: Peter Nordbeck, MD, PhD — Wuerzburg University Hospital
- Study coordinator: Peter Nordbeck, MD, PhD
- Email: nordbeck_p@ukw.de
- Phone: 004993120139181
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.