Actively Recruiting
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
Led by Mackay Memorial Hospital · Updated on 2023-11-18
150
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Fabry disease (FD) is a rare genetic disorder caused by a deficiency of the enzyme alpha-Gal A, leading to the buildup of certain lipids in the body's cells. This condition causes serious problems affecting many organs and can lead to heart issues, kidney failure, and strokes, especially in middle-aged adults. Researchers are studying various biomarkers and cardiac imaging methods to better understand disease progression and treatment effects, focusing on patients with FD, including those with a specific cardiac variant common in Taiwan. This observational study involves two groups of adults with diagnosed Fabry disease: those who have never received enzyme replacement therapy (ERT) and those treated with agalsidase beta, an ERT drug given according to local medical guidelines. Participants must show abnormalities in cardiac biomarkers, plasma Gb3 or Lyso-Gb3 levels, electrocardiography (ECG), or cardiac imaging. The study aims to develop a scoring system that uses these measures to evaluate disease severity and response to treatment. Participants will attend clinic visits for assessments including biomarker tests and cardiac imaging over a period of up to three years. Researchers will analyze which biomarkers and imaging results best indicate disease progression and how these change with treatment. The main goal is to create a comprehensive tool to monitor Fabry disease status and treatment outcomes safely and without invasive procedures like cardiac biopsy.
CONDITIONS
Brief Title
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 18 years or older
- Male or female diagnosed with Fabry disease
- Have at least one abnormal finding in cardiac biomarkers, plasma Gb3 or Lyso-Gb3, ECG, or cardiac imaging
- For Group A: no prior enzyme replacement therapy (ERT) treatment
- For Group B: previously treated or currently treated with agalsidase beta (ERT)
- Willing and able to attend clinic visits and complete study procedures
You will not qualify if you...
- Unwilling to sign informed consent form
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 3 years
Participants are observed through collection of biomarkers and cardiac imaging data to evaluate disease progression and treatment outcomes.
Regular visits as scheduled by the clinic for assessments
Trial Site Locations
Total: 1 location
1
Charles Jia-Yin Hou
Taipei, Taiwan
Actively Recruiting
Research Team
C
Charles Jia-Yin Hou
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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