Status:
COMPLETED
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Brief 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-sectiona...
Eligibility Criteria
Inclusion
- Patient
- Patients with a documented diagnosis of Fabry Disease (FD)
- Patients who have received ≥4 doses of ERT (with agalsidase alfa or agalsidase beta) for the treatment of FD.
- Patients who present to the participating hospital(s) or treatment centre(s) for administration of a dose of ERT (as part of their routine treatment) during the data collection period.
- Caregiver
- Self-identifies as a caregiver of a patient with FD for whom written informed consent has been obtained for inclusion in the study.
- Patient
Exclusion
- Patients who are unable or unwilling to give consent for study participation.
- Patients whose ERT preparation and administration takes place exclusively in the home setting with no healthcare professional (HCP) involvement in preparation of the infusion.
- For the time and motion evaluation: Patients whose ERT is administered by a HCP who does not consent to be observed.
- Caregiver
Key Trial Info
Start Date :
March 1 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 18 2022
Estimated Enrollment :
82 Patients enrolled
Trial Details
Trial ID
NCT04281537
Start Date
March 1 2020
End Date
May 18 2022
Last Update
November 4 2024
Active Locations (9)
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1
Amicus Therapeutics, Inc.
Philadelphia, Pennsylvania, United States, 19104
2
Instituto de Genética e Erros Inatos do Metabolismo (IGEIM)
São Paulo, Brazil
3
Keio University Hospital
Tokyo, Japan
4
Yokohama Municipal Citizen's Hospital
Yokohama, Japan