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ACTIVE NOT RECRUITING
NCT06052800

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

Sponsor: Sanofi

View on ClinicalTrials.gov

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.

Official title: An Observational Study of Enzyme Replacement Therapy-Naïve and Agalsidase Beta-Treated Fabry Disease Patients With GLA IVS4 919 G>A Mutation in Taiwan

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

OBSERVATIONAL

Enrollment

78

Start Date

2023-09-13

Completion Date

2026-09-10

Last Updated

2025-03-21

Healthy Volunteers

No

Conditions

Locations (5)

Investigational Site Number : 1580004

Taichung, Taiwan

Investigational Site Number : 1580005

Tainan, Taiwan

Investigational Site Number : 1580001

Taipei, Taiwan

Investigational Site Number : 1580003

Taipei, Taiwan

Investigational Site Number : 1580002

Taipei, Taiwan