Monitoring Fabry Disease with Biomarkers and Cardiac Imaging

Is this trial a fit for you?

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 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.

Why it matters

Who can join: the full rules

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 it's running

• Charles Jia-Yin Hou
  Taipei, Taiwan Recruiting

Study contacts

• Study coordinator: Charles Jia-Yin Hou
• Email: jiayinhou@gmail.com
• Phone: +886905960500

Similar studies

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

• 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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  Recruiting Institutul Clinic Fundeni · Bucharest · NCT07575347

• 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

• Using advanced heart MRI to improve diagnosis and risk prediction for rare cardiomyopathies

  This project tests whether advanced cardiac MRI techniques can help diagnose and predict future risk for people with rare heart muscle diseases like Danon, Fabry, cardiac amyloidosis, Noonan syndrome, and cardiac sarcoidosis.

  Recruiting Chinese Academy of Medical Sciences, Fuwai Hospital · Beijing · NCT07336394

• Quality of life for people 65+ with Fabry disease, treated or untreated

  This project will see if starting or continuing Fabry disease treatment changes quality of life over two and five years in people aged 65 and older.

  Recruiting Groupe Hospitalier Diaconesses Croix Saint-Simon · Paris, France · NCT07277361

• Agalsidase alfa for heart inflammation in Fabry disease measured by PET‑CMR

  This study will test whether agalsidase alfa reduces heart inflammation in people with Fabry disease by comparing PET‑CMR scans before and after 12 months of treatment.

  Recruiting Yonsei University · Seoul · NCT07235709

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.