酵素補充療法によるファブリー病の治療における患者と臨床医の両方の経験を説明する研究。

基本情報

NCT ID

NCT04281537

ステータス

完了

試験のフェーズ

-

試験タイプ

観察

目標被験者数

82

治験依頼者名

Amicus Therapeutics

概要

This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study comprised a prospective time and motion evaluation and a cross-sectional evaluation of patient and caregiver-reported outcomes. The study evaluated the time associated with the preparation and administration of a single dose of enzyme replacement therapy (ERT) in patients by health care providers as well as the impact on Fabry patients and caregivers time and costs associated with an ERT treatment. The study also evaluated the patients' quality of life wellbeing, fatigue and work productivity.

対象疾患

介入

依頼者（Sponsor）

実施施設 (2)