A Study of Migalastat in Fabry Disease

Launched by MANCHESTER UNIVERSITY NHS FOUNDATION TRUST · May 13, 2019

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Migalastat for people with Fabry disease, a rare condition that affects how the body processes certain fats, leading to problems in various organs, especially the heart. The trial aims to understand how Migalastat impacts the heart and blood vessels in patients with Fabry disease. It will help identify which patients benefit the most from this treatment and when it is best to start the medication. By using advanced, non-invasive tests, researchers hope to gain valuable insights into how this treatment can be personalized for better care.

To be eligible for this study, participants must be at least 16 years old, have a confirmed diagnosis of Fabry disease, and be starting treatment with Migalastat. Those who cannot undergo a specific heart imaging test called a cardiac MRI will not be able to participate. If you join the study, you can expect to undergo some tests that will help researchers learn more about how your heart and blood vessels are affected by Fabry disease and how they respond to Migalastat. This information could lead to improved treatment options for you and others with Fabry disease in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Confirmed Fabry disease Aged 16 or over Beginning clinical treatment with Migalastat
• Exclusion Criteria:
• • Contraindication to cardiac MRI