Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

Plain-language summary

This study evaluates the effect of Agalsidase Alfa enzyme therapy on heart inflammation in people with Fabry disease, using [18F]-FDG PET-CMR imaging. It enrolls patients aged 15 to 75 with confirmed Fabry disease and cardiac involvement who have not used Agalsidase Alfa in the last 12 months. This is a study with no assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

Yonsei University

Start date

11/12/2025

Estimated completion

11/1/2029

Countries

South Korea

Related genes

Locations

• Seoul, South Korea

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* 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
* Patients who have active malignancy
* Subject who the investigator deems inappropriate to participate in this study