CI

At a glance

ClinicalIndex Comparison Record
N/ACompleted· 82 enrolled
Drug / intervention
Enzyme Replacement Therapy (ERT)drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04281537
NCT04281537N/ACompleted

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

Amicus Therapeutics·observational·Posted Feb 24, 2020·Updated Feb 5, 2026

In Brief

An observational study evaluating Enzyme Replacement Therapy (ERT) for Fabry Disease. Completed, enrolled 82 participants across 9 sites in 5 countries.

Detailed 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.

Study Details

Study Typeobservational
Allocation--
Masking--
Primary Purpose--
ConditionsFabry Disease
CountriesBrazil, Japan, Taiwan, Turkey (Türkiye), United States
Collaborators--

Timeline

N/ACompletedFinished
2020202120222023202420252026
First PostedFeb 24, 2020
Enrollment StartMar 1, 2020
Primary CompletionMay 18, 2022
TodayJul 2, 2026
Enrollment to primary: 2.2 yearsPosted 6.4 years ago

Interventions

Enzyme Replacement Therapy (ERT)drug

ERT infusion every other week