Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences

  • End date
    Sep 23, 2022
  • participants needed
  • sponsor
    Amicus Therapeutics France SAS
Updated on 13 February 2022


In the shared decision-making process, patients should express their expectations and preferences regarding treatment to the physician. A specific questionnaire addressing needs and expectations of Fabry patients has been built at the initiative of Amicus. In addition, this questionnaire also evaluates the benefit of treatment from the patient's perspective. Nothing is known until now on patient's expectations, potential clustering of patients regarding their expectations and evaluation of treatment benefit from the patients perspective.

Study objectives are differentiated according to the study phase (inclusion and follow-up). At inclusion, the primary objective is to cluster patients according to their needs and expectations regarding treatment. During follow-up, the primary objective is to evaluate treatment benefit in relation with patients needs and expectations.


Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study. Decision to treat the patient should be made independently of the patient's participation in the study.

Condition Fabry Disease, Anderson Fabry Disease
Treatment Characterization of patients expectations and preferences regarding their treatment
Clinical Study IdentifierNCT04043273
SponsorAmicus Therapeutics France SAS
Last Modified on13 February 2022


Yes No Not Sure

Inclusion Criteria

Patients 16 years old
Diagnosed with Fabry disease
With amenable mutation
Decision by clinician to start or pursue ongoing ERT or migalastat
Non-opposition form to participate in the study signed

Exclusion Criteria

Concomitant patient participation in an interventional clinical study (Category 1 interventional research or category 2 interventional research according to the Jard law classification in France)
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
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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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