A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
Completed
Conditions studied: Fabry Disease
In brief
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.
Key facts
- Study ID
- NCT04281537
- Run by
- Amicus Therapeutics
- People needed
- 82
- Starts
- 2020-03-01
- Expected to finish
- 2022-05-18
- Last updated by the study team
- 2026-02-05
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Amicus Therapeutics, Inc. — Philadelphia, Pennsylvania, United States
- Instituto de Genética e Erros Inatos do Metabolismo (IGEIM) — São Paulo, Brazil
- Keio University Hospital — Tokyo, Japan
- Yokohama Municipal Citizen's Hospital — Yokohama, Japan
- National Taiwan University — Taipei, Taiwan
- Taipei Veterans General Hospital — Taipei, Taiwan
- Gazi University Hospital — Ankara, Turkey (Türkiye)
- Dokuz Eylul University Medical Faculty — Izmir, Turkey (Türkiye)
- Ege University Hospital — Izmir, Turkey (Türkiye)
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.