NCT04281537

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their Caregivers During the Treatment of Fabry Disease With Enzyme Replacement Therapy With Agalsidase Alfa and Agalsidase Beta

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 82 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: —

Summary

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.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Enzyme Replacement Therapy (ERT)	ERT infusion every other week

Timeline

• Start date: 2020-03-01
• Primary completion: 2022-05-18
• Completion: 2022-05-18
• First posted: 2020-02-24
• Last updated: 2026-02-05
• Results posted: 2024-11-04

Locations

9 sites across 5 countries: United States, Brazil, Japan, Taiwan, Turkey (Türkiye)