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

Study Overview
=================
Brief Summary
-----------------
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.

Detailed Description
-----------------
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.

Official Title
-----------------
A French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat - the MIGA-FAB Study

Conditions
-----------------
Fabry Disease

Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Minimum Age: 16 Years

Sexes Eligible for Study
-----------------
All

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Miga-Fab patients<br>Miga-Fab is a French prospective, observational cohort study of patients with Fabry disease treated with migalastat | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety - SAEs | Evaluation of the occurrence ie the number of SAEs | up to 60 months | 
| Safety - vital signs | Evaluation of resting blood pressure | up to 60 months | 
| Effectiveness - Fabry Associated Clinical Events (FACEs) | Evaluation of the occurrence of the FACEs ie total number of cardiac, cerebrovascular, and renal events | up to 60 months | 
| Effectiveness - survival | Survival among all patients enrolled, as assessed by recorded patient death from any cause | From date of inclusion until the date of death from any cause, assessed up to 60 months | 
| SF-12 12-Item Short Form Health Survey | Evaluation of QOL by the 12-Item Short Form Health Survey (SF-12) ; the higher the score the worse the quality of life is | up to 60 months | 
| BPI | Brief pain inventory questionnaire ; the higher the score the more intense the pain is | up to 60 months | 
| FABPRO-GI | Fabry Disease Patient-Reported Outcome-Gastro intestinal Signs and Symptoms Questionnaire ; the higher the score the more importante the GI symptoms are | inclusion to last visit | 
| Cardiac echo imagery | Echocardiogram (Echo) Left Ventricular Mass Index (LVMI) | up to 60 months | 
| Treatment compliance | Patient adherence evaluation (% of taken intakes per month) as reported monthly through self-reports of forgotten intakes by the patient | up to 60 months |