Skip to main content
OpenTrials
Completed

NCT Number: NCT04602364

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

16 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

CHU Angers, Angers, France

Loading trial locations.

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Noninterventional

Other

Not applicable; Noninterventional study

Primary outcomes

  1. Safety - SAEs

    Time frame: up to 60 months

    Evaluation of the occurrence ie the number of SAEs

  2. Safety - vital signs

    Time frame: up to 60 months

    Evaluation of resting blood pressure

  3. Effectiveness - Fabry Associated Clinical Events (FACEs)

    Time frame: up to 60 months

    Evaluation of the occurrence of the FACEs ie total number of cardiac, cerebrovascular, and renal events

  4. Effectiveness - survival

    Time frame: From date of inclusion until the date of death from any cause, assessed up to 60 months

    Survival among all patients enrolled, as assessed by recorded patient death from any cause

  5. SF-12 12-Item Short Form Health Survey

    Time frame: up to 60 months

    Evaluation of QOL by the 12-Item Short Form Health Survey (SF-12) ; the higher the score the worse the quality of life is

  6. BPI

    Time frame: up to 60 months

    Brief pain inventory questionnaire ; the higher the score the more intense the pain is

  7. FABPRO-GI

    Time frame: inclusion to last visit

    Fabry Disease Patient-Reported Outcome-Gastro intestinal Signs and Symptoms Questionnaire ; the higher the score the more importante the GI symptoms are

  8. Cardiac echo imagery

    Time frame: up to 60 months

    Echocardiogram (Echo) Left Ventricular Mass Index (LVMI)

  9. Treatment compliance

    Time frame: up to 60 months

    Patient adherence evaluation (% of taken intakes per month) as reported monthly through self-reports of forgotten intakes by the patient

Sponsors and collaborators

Lead sponsor

Amicus Therapeutics France SAS

Industry

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Oct 26, 2020
Registry last updated
May 23, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.