Status:
ACTIVE_NOT_RECRUITING
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
Lead Sponsor:
Sanofi
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Brief Summary
This is a national, multicenter, observational, cohort study designed to assess clinical outcomes upon agalsidase beta treatment, to characterize the clinical manifestations, and to collect the natura...
Detailed Description
Study Design Time Perspective: Retrospective and Prospective
Eligibility Criteria
Inclusion
- \- Provide signed informed consent.
- Cohort 1:
- Male or female Fabry disease patient with documented GLA IVS4 in medical record.
- Age ≥ 18 years old at the time of signing informed consent.
- The maximum proportion of female is 20% of cohort 1.
- Patient who has received agalsidase beta treatment for at least 6 months.
- The data of LVMI, LPWT, IVST and blood lyso-Gb3 concentration are all available within 6 months prior to agalsidase beta treatment initiation.
- Cohort 2:
- Male or female Fabry disease patient with documented GLA IVS4 in medical record.
- Age ≥ 18 years old at the time of signing informed consent.
- The maximum proportion of female is 20% of cohort 2.
- Patient who plans to apply for the National Health Insurance Reimbursement for agalsidase beta medication.
- The data of LVMI, LPWT, IVST and blood lyso-Gb3 concentration are all available within 6 months prior to agalsidase beta treatment initiation.
- Cohort 3:
- Male or female Fabry disease patients with documented GLA IVS4 mutation in medical record.
- Male patient is aged ≥ 30 years old and female patient is aged ≥ 40 years old at the time of signing informed consent.
- The maximum proportion of female is 20% of cohort 3.
- Patient who has never received agalsidase alpha or agalsidase beta treatment (ERT-naïve).
- Elevated blood lyso-Gb3.
- At least ONE of the following conditions documented in medical record:
- cardiac parameter abnormalities (e.g. via imaging, electrophysiology, or biomarker);
- at least one FD-related sign/symptom.
- The data of LVMI, LPWT, IVST and blood lyso-Gb3 concentration are all available within 6 months.
- Patients who are expected not to receive ERT or FD-specific treatment per investigator's judgement.
Exclusion
- Any condition that, in the opinion of the Investigator, may interfere with patient's participation in the study, such as life expectancy of less than 6 months (e.g. diagnosed with malignancy, CAD)
- Fabry patients who have severe heart disease (NYHA Class IV) or severe myocardial fibrosis per investigator judgement
- Known non-Fabry disease infiltrative cardiomyopathy including amyloidosis
- Known non-GLA genetic (e.g., sarcomeric, metabolic mutations) hypertrophic cardiomyopathy.
- Patients who are receiving any Fabry disease-specific treatment (enzyme replacement therapy, chaperone therapy, substrate reduction therapy, or gene therapy) other than agalsidase beta for Fabry disease
- Pregnancy or suspected pregnancy
- Patient diagnosed with moderate to severe dementia
- Unstable patient condition as judged by investigator (e.g., hypertension, diabetes, and systematic disease)
Key Trial Info
Start Date :
September 13 2023
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 10 2026
Estimated Enrollment :
78 Patients enrolled
Trial Details
Trial ID
NCT06052800
Start Date
September 13 2023
End Date
September 10 2026
Last Update
March 21 2025
Active Locations (5)
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1
Investigational Site Number : 1580004
Taichung, Taiwan, 407219
2
Investigational Site Number : 1580005
Tainan, Taiwan, 704
3
Investigational Site Number : 1580001
Taipei, Taiwan, 100
4
Investigational Site Number : 1580003
Taipei, Taiwan, 104