NCT03683966

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

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

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 30 (estimated)
• Sponsor: Wuerzburg University Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: —

Summary

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

Detailed 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.

Conditions

• Rare Diseases
• Fabry Disease
• Adherence, Medication
• Quality of Life

Timeline

• Start date: 2017-10-27
• Primary completion: 2023-12-31
• Completion: 2023-12-31
• First posted: 2018-09-25
• Last updated: 2022-07-11

Locations

1 site across 1 country: Germany