Monitoring Fabry Disease with Biomarkers and Cardiac Imaging
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
This study is testing how well heart scans and blood tests can help track the progress of Fabry disease and see how patients respond to treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 150 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Mackay Memorial Hospital Academic / other |
| Locations | 1 site (Taipei) |
| Trial ID | NCT05698901 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on patients diagnosed with Fabry disease, a rare genetic disorder that leads to severe multi-systemic complications. It aims to monitor disease progression and therapy response through the assessment of cardiac biomarkers and imaging techniques. Patients will be categorized into two groups: those who are treatment-naïve and those who have previously received enzyme replacement therapy. The study seeks to identify early signs of cardiac involvement to facilitate timely intervention.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older diagnosed with Fabry disease who exhibit specific cardiac abnormalities.
Not a fit: Patients who are unwilling to sign the informed consent form will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved monitoring and earlier intervention for patients with Fabry disease, potentially reducing the risk of severe complications.
How similar studies have performed: While there have been studies on Fabry disease, this specific approach utilizing biomarkers and cardiac imaging for monitoring is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 18 years or older * Male or female with Fabry disease diagnosed * Presence of any one of following abnormal criteria of either: 1) Cardio-specific Biomarker; 2) Abnormal elevated value of plasma Gb3 or Lyso-Gb3; 3) Electrocardiography (ECG); 4) Cardio-specific Image. * Group A: ERT Treatment naïve Fabry patients * Group B: Agalsidase beta (ERT) exposed or treated Fabry patients * Willing and able to comply with the required clinic visits, study procedures and assessments Exclusion Criteria: * Patient who are unwilling to sign inform consent form
Where this trial is running
Taipei
- Charles Jia-Yin Hou — Taipei, Taiwan (Recruiting)
Study contacts
- Study coordinator: Charles Jia-Yin Hou
- Email: jiayinhou@gmail.com
- Phone: +886905960500
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.