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

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

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.

Study Overview

• Status: Completed
• Conditions: Fabry Disease
• Intervention / Treatment: Other: Noninterventional

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.

• Study Type: Observational
• Enrollment (Actual): 48

Contacts and Locations

Study Locations

• France
  • Angers, France
    • CHU Angers
  • Bordeaux, France
    • CHU de Bordeaux
  • Bordeaux, France
    • Hôpital Pellegrin
  • Caen, France
    • CHU Caen
  • Lille, France
    • Chu Lille
  • Lyon, France
    • Hôpital Femme Mère Enfant
  • Marseille, France
    • Hôpital de la Conception | AP-HM
  • Nancy, France
    • CHU de Nancy
  • Nantes, France
    • CHU Nantes Hôtel Dieu
  • Paris, France
    • Hôpital Tenon AP-HP
  • Paris, France
    • Hôpital de la Croix Saint Simon
  • Strasbourg, France
    • Hôpitaux Universitaires de Strasbourg
  • Toulouse, France
    • CHU TOULOUSE Rangueil

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

• 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.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Miga-Fab patients; Miga-Fab is a French prospective, observational cohort study of patients with Fabry disease treated with migalastat	Other: Noninterventional; Not applicable; Noninterventional study

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

Collaborators and Investigators

• Sponsor: Amicus Therapeutics France SAS
• Investigators: Principal Investigator: Olivier Lidove, Dr., Groupe Hospitalier Diaconnesses Croix Saint Simon

Study record dates

Study Major Dates

• Study Start (Actual): October 15, 2020
• Primary Completion (Actual): June 30, 2023
• Study Completion (Actual): June 30, 2023

Study Registration Dates

• First Submitted: January 22, 2020
• First Submitted That Met QC Criteria: October 20, 2020
• First Posted (Actual): October 26, 2020

Study Record Updates

• Last Update Posted (Actual): May 23, 2024
• Last Update Submitted That Met QC Criteria: May 22, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Metabolic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Fabry Disease
• Other Study ID Numbers: MIGA-FAB

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No