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

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04602364
• Other study ID #: MIGA-FAB
• Status: Completed
• Start date: October 15, 2020
• Est. completion date: June 30, 2023

Study information

• Verified date: May 2024
• Source: Amicus Therapeutics France SAS
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 48
• Est. completion date: June 30, 2023
• Est. primary completion date: June 30, 2023
• Gender: All
• Age group: 16 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Other:
• Noninterventional
Not applicable; Noninterventional study

Locations

Country	Name	City	State
France	CHU Angers	Angers
France	CHU de Bordeaux	Bordeaux
France	Hôpital Pellegrin	Bordeaux
France	CHU Caen	Caen
France	CHU Lille	Lille
France	Hôpital femme mère enfant	Lyon
France	Hôpital de la Conception | AP-HM	Marseille
France	CHU de Nancy	Nancy
France	CHU Nantes Hôtel Dieu	Nantes
France	Hôpital de la Croix Saint Simon	Paris
France	Hôpital Tenon AP-HP	Paris
France	Hôpitaux Universitaires de Strasbourg	Strasbourg
France	CHU Toulouse Rangueil	Toulouse

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics France SAS

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety - SAEs	Evaluation of the occurrence ie the number of SAEs	up to 60 months
Primary	Safety - vital signs	Evaluation of resting blood pressure	up to 60 months
Primary	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
Primary	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
Primary	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
Primary	BPI	Brief pain inventory questionnaire ; the higher the score the more intense the pain is	up to 60 months
Primary	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
Primary	Cardiac echo imagery	Echocardiogram (Echo) Left Ventricular Mass Index (LVMI)	up to 60 months
Primary	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