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

NCT04281537 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 82

Last updated 2026-02-05

Study results available
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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

Interventions

DRUG

Enzyme Replacement Therapy (ERT)

ERT infusion every other week

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-03-01
Primary Completion
2022-05-18
Completion
2022-05-18

Countries

  • United States
  • Brazil
  • Japan
  • Taiwan
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Diseases

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 NCT04281537 on ClinicalTrials.gov