About this 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.
Eligibility criteria
Qualifiers
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
Disqualifiers
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 are pregnant
Trial design
Treatments tested in this trial
- Not listed