ClinicalTrials.gov record NCT03362164
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a prospective observational cohort study of adults with genetically confirmed Fabry disease who provide informed consent. It collects long-term clinical data to identify which features are associated with serious cardiac events, including arrhythmias, sudden cardiac death, and terminal heart failure (death or heart transplantation). The study records cardiac events and related outcomes over time, with events followed through 2032.
What it is studying. The study is testing which clinical factors predict the incidence of cardiac arrhythmias and sudden cardiac death, and which predict death or the need for heart transplantation from terminal heart failure in people with Fabry disease. It records outcomes such as cardiac death, malignant arrhythmias, and heart transplantation.
What the researchers hope to learn. Estimate the risk of cardiac arrhythmias and sudden cardiac death in patients with Fabry disease, and estimate the risk of death or heart transplantation due to terminal heart failure.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This study evaluates predictors for the incidence of arrhythmias and sudden cardiac death as well as terminal heart failure in patients with Fabry disease.
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Fabry disease (genetically confirmed)
- Signed informed consent
- 18 years and older
Exclusion criteria
- No informed consent
- Withdrawal of informed consent
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants sign informed consent and are followed over time as part of a prospective observational study. The study collects clinical information about heart-related outcomes (arrhythmias, heart transplantation, cardiac death) and documents events through routine care and follow-up; outcomes are tracked through to 2032 as listed in the registry. No treatments are assigned by the study. The registry record does not specify additional invasive procedures beyond routine clinical care.
How the study is structured. A prospective observational cohort: participants are followed over time and their clinical events are recorded. No treatments are assigned by the study. Cardiac events and outcomes are tracked and documented through the study period (events collected up to the year 2032).
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Germany. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Fabry disease
Important dates
- Study start
- January 2001
- Primary completion
- March 2032 (estimated)
- Study completion
- March 2032 (estimated)
- Target enrollment
- 650 participants (estimated)
- Last updated on registry
- Jul 7, 2022
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Wuerzburg University Hospital
- Principal investigator
- PD Dr. Peter Nordbeck, Head Consultant Cardiology and Chief of Electrophysiology
Wuerzburg University Hospital - Collaborators
- Competence Network Heart Failure
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
- St George's, University of London
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Peter Nordbeck, MD, PhD 📞 004993120139181 ✉ nordbeck_p@ukw.de
- Jonas Muentze, MD 📞 004993120139958 ✉ muentze_j@ukw.de
Participating sites (1)
- Wuerzburg University HospitalRecruiting
Würzburg, Bavaria, 97080 · Germany
- Irina Schumacher · 004993120139714 · schumacher_i@ukw.de
Terms used on this page · explanations by MedicalUpdates.ai
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Natural history
- A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.
- prospective cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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