Clinical trial Recruiting at 1 site Observational Ages 18 and up

Evaluating heart involvement in patients with Fabry disease

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

Run by Wuerzburg University Hospital · NCT03362164 · Data updated 2026-11-12

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 typeObservational
Ages18 Years and up
SexAll
Enrollment650 (estimated)
SponsorWuerzburg University Hospital
Academic / other
Locations1 site (Würzburg, Bavaria)
StatusRecruiting
StartedJanuary 2001
Main results expectedMarch 2032
Study endsMarch 2032
Registry record updatedJuly 7, 2022
Trial IDNCT03362164 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Ideal candidates for this study are adults aged 18 and older with genetically confirmed Fabry disease.

Probably not a fit

Patients who do not provide informed consent or withdraw consent will not benefit from this study.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

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 gone: While the study addresses a rare condition, similar observational studies have shown success in understanding cardiac implications in other lysosomal storage disorders.

Who can join: the full rules

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 it's running

  • 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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  • Lucerastat in untreated adult men with Fabry disease shows how well it works and safety

    This study tests lucerastat to see if it can reduce Gb3 buildup and be safe in untreated adult men with Fabry disease.

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  • Socio‑professional inclusion for young people (15–25) with childhood-onset rare genetic disabilities

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    Recruiting Imagine Institute · Paris, Île-de-France Region · NCT07527624

  • Anderson-Fabry disease fitness improvement program

    This study tests whether a 12-week physiotherapy-led exercise program can improve function, fitness, strength, and quality of life in adults with Fabry disease.

    Recruiting General University Hospital, Prague · Prague · NCT07495410

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.