NCT04602364

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
48

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Oct 2020

Typical duration for all trials

Geographic Reach
1 country

13 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 22, 2020

Completed
9 months until next milestone

Study Start

First participant enrolled

October 15, 2020

Completed
11 days until next milestone

First Posted

Study publicly available on registry

October 26, 2020

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2023

Completed
Last Updated

May 23, 2024

Status Verified

May 1, 2024

Enrollment Period

2.7 years

First QC Date

January 22, 2020

Last Update Submit

May 22, 2024

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (9)

  • 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

Study Arms (1)

Miga-Fab patients

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

Other: Noninterventional

Interventions

NoninterventionalOTHER

Not applicable; Noninterventional study

Miga-Fab patients

Eligibility Criteria

Age16 Years+
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

The Miga-Fab is open to participants with Fabry disease treated by migalastat. There is no predetermined sample size.

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

CHU Angers

Angers, France

Location

CHU de Bordeaux

Bordeaux, France

Location

Hôpital Pellegrin

Bordeaux, France

Location

CHU Caen

Caen, France

Location

CHU Lille

Lille, France

Location

Hôpital femme mère enfant

Lyon, France

Location

Hôpital de la Conception | AP-HM

Marseille, France

Location

CHU de Nancy

Nancy, France

Location

CHU Nantes Hôtel Dieu

Nantes, France

Location

Hôpital de la Croix Saint Simon

Paris, France

Location

Hôpital Tenon AP-HP

Paris, France

Location

Hôpitaux Universitaires de Strasbourg

Strasbourg, France

Location

CHU Toulouse Rangueil

Toulouse, France

Location

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersVascular DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Study Officials

  • Olivier Lidove, Dr.

    Groupe Hospitalier Diaconnesses Croix Saint Simon

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 22, 2020

First Posted

October 26, 2020

Study Start

October 15, 2020

Primary Completion

June 30, 2023

Study Completion

June 30, 2023

Last Updated

May 23, 2024

Record last verified: 2024-05

Data Sharing

IPD Sharing
Will not share

Locations

FR(13)