Agalsidase Alfa and Heart Inflammation in People With Fabry Disease
Studies heart inflammation in people with Fabry disease receiving agalsidase alfa over 12 months.
Plain-language title generated from the registry record
Registry title: Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study
Key facts
- Where
- Seoul, South KoreaSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 15 and 75 years old?
Source: NCT07235709, who can take part
Where in South Korea
- Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of MedicineSeoul, South KoreaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Yonsei University
- Trial code
- NCT07235709
- Record updated
- 1 December 2025
- Original description
This is a prospective observational study. All patients will initiate and maintain treatment with agalsidase alfa during the study period. All patients will receive a full standard of care concomitant medication for the treatment of their cardiac condition. Twenty-five patients with genetically confirmed Anderson-Fabry disease will undergo PET-CMR at baseline and after 12 months of treatment with Agalsidase Alfa for follow-up.Read full original descriptionShow less
Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study (ESCAPE-FABRY Trial)
This is a prospective observational study. All patients will initiate and maintain treatment with agalsidase alfa during the study period. All patients will receive a full standard of care concomitant medication for the treatment of their cardiac condition. Twenty-five patients with genetically confirmed Anderson-Fabry disease will undergo PET-CMR at baseline and after 12 months of treatment with Agalsidase Alfa for follow-up.
Inclusion: Patients aged 15-75 years with Fabry disease confirmed by enzyme assay and gene test; Patients have not undergone ERT for more than 12 months or patients have not used Agalsidase Alfa in the last 12 months; Patients who have cardiac involvement of Fabry disease (end diastolic maximal wall thickness ≥ 12mm on echocardiography or CMR, decreased native T1 value on CMR, unexplained distinct diastolic dysfunction, unexplained decreased global longitudinal strain on 2D strain echocardiography, or biopsy-proven cardiac involvement); Patients provided written informed consent to participate in this study; The patient, or patient's legally authorized representative(s), if applicable, understands the nature, scope, and possible consequences of the study and has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee; The patient must be sufficiently cooperative to participate in this clinical study as judged by the investigator.
Exclusion: Contraindication for enzyme replacement treatment with Agalsidase Alfa; Patients have previously been treated with Agalsidase Alfa for > 12 months; Patients unable to undergo PET-CMR due to any condition; Patients who have active malignancy; Patients who are pregnant; Subject who the investigator deems inappropriate to participate in this study.