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Clinical Trial Summary

The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.


Clinical Trial Description

This will be a cross-sectional study conducted in approximately 130 individuals (or representative parents/caregivers of patients) living with Fabry disease. All study participants will complete the RSVP followed by a structured interview conducted by trained interviewers. It is estimated that each respondent will need up to 60 minutes for the entire process; 10 minutes to complete the RSVP including uploading the proof of Fabry disease diagnosis or verifying membership with Fabry groups, including but not limited to: Fabry Support and Information Group, National Fabry Disease Foundation, MPS Society UK, Morbus Fabry Selbsthilfergruppe, Fabry International Network, or others, and approximately 50 minutes to complete the interview. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04804566
Study type Observational
Source Amicus Therapeutics
Contact
Status Completed
Phase
Start date March 1, 2023
Completion date November 1, 2023

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