Trials / Active Not Recruiting
Active Not RecruitingNCT06052800
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
An Observational Study of Enzyme Replacement Therapy-Naïve and Agalsidase Beta-Treated Fabry Disease Patients With GLA IVS4 919 G>A Mutation in Taiwan
- Status
- Active Not Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 78 (actual)
- Sponsor
- Sanofi · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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 natural history on male and female Fabry disease adult patients who carry the GLA IVS4. This study aims to retrospectively and prospectively investigate the disease natural history, clinical manifestations, and the treatment outcomes upon agalsidase beta in Fabry disease (FD) patients carrying the GLA IVS4 mutation from medical records, physician assessments, and patient-reported outcomes.
Detailed description
Study Design Time Perspective: Retrospective and Prospective
Conditions
Timeline
- Start date
- 2023-09-13
- Primary completion
- 2026-09-10
- Completion
- 2026-09-10
- First posted
- 2023-09-25
- Last updated
- 2025-03-21
Locations
5 sites across 1 country: Taiwan
Source: ClinicalTrials.gov record NCT06052800. Inclusion in this directory is not an endorsement.