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

NCT06052800 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 78

Last updated 2026-05-15

No results posted yet for this study

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.

Conditions

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences and Operations · Sanofi

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-09-13
Primary Completion
2026-09-10
Completion
2026-09-10

Countries

  • Taiwan

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06052800 on ClinicalTrials.gov