Clinical trial NCT04602364: French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat
Official title: A French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat - the MIGA-FAB Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Fabry Disease
Lead sponsor: Amicus Therapeutics France SAS
Sponsor class: INDUSTRY
First posted: 2020-10-26
Enrollment: 48
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The Miga-Fab is open to participants with Fabry disease treated by migalastat. There is no predetermined sample size.

Brief summary: This is a noninterventional 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.

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

Primary outcomes:
measure: Safety - SAEs
timeFrame: up to 60 months
measure: Safety - vital signs
timeFrame: up to 60 months
measure: Effectiveness - Fabry Associated Clinical Events (FACEs)
timeFrame: up to 60 months
measure: Effectiveness - survival
timeFrame: From date of inclusion until the date of death from any cause, assessed up to 60 months
measure: SF-12 12-Item Short Form Health Survey
timeFrame: up to 60 months
measure: BPI
timeFrame: up to 60 months
measure: FABPRO-GI
timeFrame: inclusion to last visit
measure: Cardiac echo imagery
timeFrame: up to 60 months
measure: Treatment compliance
timeFrame: up to 60 months
Secondary outcomes:
Other outcomes:
Locations:
city: Angers
country: France
facility: CHU Angers
city: Bordeaux
country: France
facility: CHU de Bordeaux
city: Bordeaux
country: France
facility: Hôpital Pellegrin
city: Caen
country: France
facility: CHU Caen
city: Lille
country: France
facility: CHU Lille
city: Lyon
country: France
facility: Hôpital femme mère enfant
city: Marseille
country: France
facility: Hôpital de la Conception | AP-HM
city: Nancy
country: France
facility: CHU de Nancy
city: Nantes
country: France
facility: CHU Nantes Hôtel Dieu
city: Paris
country: France
facility: Hôpital de la Croix Saint Simon
city: Paris
country: France
facility: Hôpital Tenon AP-HP
city: Strasbourg
country: France
facility: Hôpitaux Universitaires de Strasbourg
city: Toulouse
country: France
facility: CHU Toulouse Rangueil
Study officials:
affiliation: Groupe Hospitalier Diaconnesses Croix Saint Simon
name: Olivier Lidove, Dr.
role: PRINCIPAL_INVESTIGATOR
References: