NCT07235709Recruiting
Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study
Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study (ESCAPE-FABRY Trial)
Source: ClinicalTrials.gov · Last updated 1 Dec 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 25 (estimated)
- First submitted
- 14 Nov 2025
- Start date
- 12 Nov 2025
- Primary completion date
- 30 Jun 2029
- Lead sponsor
- Yonsei University
- Diseases
- Fabry disease
- NCT number
- NCT07235709
What you commit to
- Study type: Observational
- Planned participants: 25
- From 12 Nov 2025 to the primary completion date, 30 Jun 2029
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Contraindication for enzyme replacement treatment with Agalsidase Alfa
- Patients have previously been treated with Agalsidase Alfa for \> 12 months
Other illness
- Patients unable to undergo PET-CMR due to any condition
- Patients who have active malignancy
Other
- Patients who are pregnant
- Subject who the investigator deems inappropriate to participate in this study
Who the study is looking for
- 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.
Age in the registry: 15 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine Coordinator of this site? Confirm its status | Seoul | South Korea | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.