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

Amicus Therapeutics

Status: Completed

Conditions: Fabry Disease

Treatments: Other: Noninterventional

Study type: Observational

Funder types: Industry

Identifiers

NCT04602364

MIGA-FAB

Details and patient eligibility

About

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.

Full 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.

Enrollment: 48 patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

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

Trial design

48 participants in 1 patient group

Miga-Fab patients

Description:

Miga-Fab is a French prospective, observational cohort study of patients with Fabry disease treated with migalastat

Treatment:

Other: Noninterventional