French Prospective Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat

    Not Recruiting
  • End date
    Jun 25, 2023
  • participants needed
  • sponsor
    Amicus Therapeutics France SAS
Updated on 8 August 2022


This is a cohort study to evaluate the effects of migalastat, on long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease.


Occurrence of key indicators of safety and effectiveness will be evaluated, such as cardiac, cerebrovascular and renal events, and overall survival. The study is designed to provide effectiveness and safety data by Q2 2023 which will cover a period up to 5 years after the migalastat launch date.

This will involve a retrospective data collection up to migalastat initiation (for patients already receiving migalastat) and a prospective follow-up from 1 to 3.5 years (depending on the time of enrollment) in migalastat-treated patients with Fabry disease who have a GLA mutation amenable to migalastat.

All visits will be scheduled and conducted according to the clinical site's standard of care. Standard of care is defined as a diagnostic and customary clinical treatment/practice process that a clinician chooses according to their clinical judgement for a Fabry disease patient. There are no study- required visits, tests or clinical assessments.

Condition Fabry's Disease, Fabry Disease, Fabry Disease
Clinical Study IdentifierNCT04602364
SponsorAmicus Therapeutics France SAS
Last Modified on8 August 2022

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