Calendario›SNY›Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
Ensayo de Sanofi en Fabry Disease (del registro, en inglés).
- Fase
- no aplica
- Estado
- En marcha, sin reclutar
- Participantes
- 78
- Centros
- 5
- Fin del objetivo primario
- sep 2026
previstos
estimado por el promotor
Qué significa cada fase y cada estado.
Estudio observacional. Comenzó en sep 2023.
Qué mide
Cohort 1: change of Left ventricular mass index (LVMI) as measured by echocardiography in agalsidase beta-treated patients (through study completion with a minimum of 2 years) (del registro, en inglés)
Cómo lo describe el promotor
En el documentoEn inglés, del registroThis 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.
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Ficha completa en ClinicalTrials.gov (NCT06052800), actualizada en may 2026.