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Clinical Trial Summary

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.


Clinical Trial Description

Prospective, longitudinal, non-comparative, open-label, multicentre, non interventional cohort study. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04043273
Study type Observational
Source Amicus Therapeutics France SAS
Contact
Status Completed
Phase
Start date April 4, 2019
Completion date January 30, 2023

See also
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