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

  • days left to enroll
  • participants needed
  • sponsor
    Amicus Therapeutics
Updated on 24 March 2022
replacement therapy
agalsidase beta
agalsidase alfa


This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study will comprise a prospective time and motion evaluation and a cross-sectional evaluation of patient and caregiver-reported outcomes. The study will evaluate the time associated with the preparation and administration of a single dose of 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 will also evaluate the patients' quality of life wellbeing, fatigue and work productivity.

Condition Fabry Disease
Treatment agalsidase beta, Agalsidase Alpha
Clinical Study IdentifierNCT04281537
SponsorAmicus Therapeutics
Last Modified on24 March 2022


Yes No Not Sure

Inclusion Criteria

Patients with a documented diagnosis of 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

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
Caregiver Exclusion Criteria
setting with no 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 (and/or the patient with FD whom they support or care for) is unable or
unwilling to give consent for study participation
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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