- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04602364
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
Study Overview
Status
Conditions
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
Enrollment (Anticipated)
Contacts and Locations
Study Locations
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Angers, France
- CHU Angers
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Bordeaux, France
- Hôpital Pellegrin
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Boulogne-sur-Mer, France
- Centre Hospitalier de Boulogne Sur Mer
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Caen, France
- CHU Caen
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Dijon, France
- Hôpital François Mitterrand
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Grenoble, France
- CHU de Grenoble site Nord, Hôpital Albert Michallon
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Lille, France
- CHU Lille
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Lyon, France
- Hôpital Femme Mère Enfant
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Marseille, France
- Hôpital de la Conception | AP-HM
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Nantes, France
- CHU Nantes Hôtel Dieu
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Paris, France
- Hôpital Tenon AP-HP
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Paris, France
- Hopital de la Croix Saint Simon
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Strasbourg, France
- Hôpitaux Universitaires de Strasbourg
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Toulouse, France
- CHU Toulouse Rangueil
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Vannes, France
- CH Bretagne Atlantique
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
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?
Design Details
Cohorts and Interventions
Group / Cohort |
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Miga-Fab patients
Miga-Fab is a French prospective, observational cohort study of patients with Fabry disease treated with migalastat
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Safety - SAEs
Time Frame: up to 60 months
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Evaluation of the occurrence ie the number of SAEs
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up to 60 months
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Safety - vital signs
Time Frame: up to 60 months
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Evaluation of resting blood pressure
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up to 60 months
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Effectiveness - Fabry Associated Clinical Events (FACEs)
Time Frame: up to 60 months
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Evaluation of the occurrence of the FACEs ie total number of cardiac, cerebrovascular, and renal events
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up to 60 months
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Effectiveness - survival
Time Frame: From date of inclusion until the date of death from any cause, assessed up to 60 months
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Survival among all patients enrolled, as assessed by recorded patient death from any cause
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From date of inclusion until the date of death from any cause, assessed up to 60 months
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SF-12 12-Item Short Form Health Survey
Time Frame: up to 60 months
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Evaluation of QOL by the 12-Item Short Form Health Survey (SF-12) ; the higher the score the worse the quality of life is
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up to 60 months
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BPI
Time Frame: up to 60 months
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Brief pain inventory questionnaire ; the higher the score the more intense the pain is
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up to 60 months
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FABPRO-GI
Time Frame: inclusion to last visit
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Fabry Disease Patient-Reported Outcome-Gastro intestinal Signs and Symptoms Questionnaire ; the higher the score the more importante the GI symptoms are
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inclusion to last visit
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Cardiac echo imagery
Time Frame: up to 60 months
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Echocardiogram (Echo) Left Ventricular Mass Index (LVMI)
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up to 60 months
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Treatment compliance
Time Frame: up to 60 months
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Patient adherence evaluation (% of taken intakes per month) as reported monthly through self-reports of forgotten intakes by the patient
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up to 60 months
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Olivier Lidove, Dr., Groupe Hospitalier Diaconnesses Croix Saint Simon
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ANTICIPATED)
Study Completion (ANTICIPATED)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
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
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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