Effect of Migalastat on Cardiac Involvement in Fabry Disease

CompletedOBSERVATIONAL
Enrollment

18

Participants

Timeline

Start Date

January 10, 2018

Primary Completion Date

January 22, 2021

Study Completion Date

January 22, 2021

Conditions
Fabry DiseaseHeart Diseases
Interventions
DIAGNOSTIC_TEST

Cardiological evaluation

"Baseline evaluation~* FAbry STabilization indEX (FASTEX)~* 12 leads ECG~* Blood samples for microRNA, TnT HS and NT-proBNP dosages~* 2D echocardiogram~* Cardio-pulmonary test~* Contrast-enhanced CMR including:~ * Cine images~ * T2 mapping sequences~ * T1 mapping sequences before and 15' after contrast medium administration~ * Late Gadolinium Enhancement (LGE) imaging~ * Phase contrast images (LVOT, aortic flow)~Follow up evaluation~•After 18 months, the same procedures will be repeated"

Trial Locations (1)

20097

IRCCS Policlinico San Donato, San Donato Milanese

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Amicus Therapeutics

INDUSTRY

collaborator

Institute of Biomedicine and Molecular Immunology - CNR

UNKNOWN

lead

Ospedale San Donato

OTHER