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MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study — MALTA-FABRY  

Quality of Life Clinical Trial

Official title:
A Structured Survey Among Fabry Patients With a Focus on Adherence to Therapy, Quality of Life and Pain Control

Clinical Trial Summary

This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.

Clinical Trial Description

Fabry disease is a rare disease and part of the group of lysosomal storage disorders. Since 2016, chaperone therapy as a new therapeutic approach is available. This study is a prospective cohort study and observes patients under therapy with migalastat. This study is suggested to help estimating the adherence of the oral therapy. All patients in treatment with migalastat in the Fabry Center Wuerzburg (FAZiT) and selected patients of other cooperating Fabry Centers are included in this study if informed consent is provided. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03683966
Study type Observational
Source Wuerzburg University Hospital
Contact Peter Nordbeck, MD, PhD
Phone 004993120139181
Email nordbeck_p@ukw.de
Status Recruiting
Phase
Start date October 27, 2017
Completion date December 31, 2023
View Details
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