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

Amicus Therapeutics

Status: Completed

Conditions: Fabry Disease

Treatments: Drug: Enzyme Replacement Therapy (ERT)

Study type: Observational

Funder types: Industry

Identifiers

NCT04281537

AT-NIS-0001

Details and patient eligibility

About

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.

Enrollment: 82 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Patient Inclusion Criteria:

• 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 Inclusion Criteria:

-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 Criteria:

• 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 Exclusion Criteria:

- Caregiver (and/or the patient with FD whom they support or care for) is unable or unwilling to give consent for study participation.

Trial design

82 participants in 2 patient groups

Patient with Fabry Disease on Enzyme Replacement Therapy (ERT)

Description:

Patients with Fabry Disease receiving Enzyme Replacement Therapy (agalsidase alfa or agalsidase beta)

Treatment:

Drug: Enzyme Replacement Therapy (ERT)

Caregiver

Description:

Caregiver of patient with Fabry Disease on Enzyme Replacement Therapy (ERT)