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

  • End date
    Jun 25, 2023
  • participants needed
  • sponsor
    Amicus Therapeutics France SAS
Updated on 25 January 2021


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 on25 January 2021


Yes No Not Sure

Inclusion Criteria

Patients with Fabry disease aged 16 years or older
eGFRCKD-EPI > 30 mL/min/1.73 m2
treated with migalastat, or who are starting migalastat upon enrollment
Patients with Fabry disease and/or parents/guardians (when applicable) who are able to understand and have provided a signed non-opposition form
Equipped with a web connection via a computer or tablet

Exclusion Criteria

Patients who are participating in a clinical trial of any investigational medicinal product or device at the time of enrollment
Patients already included in the present study
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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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