MedTrace Logo

A Study of Migalastat in Fabry Disease

NCT03949920 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 21

Last updated 2023-05-31

No results posted yet for this study

Summary

Fabry disease is a rare metabolic condition characterised by the widespread deposition of sphingolipids in multiple organ systems. Cardiac involvement is common, it occurs in fifty percent of patients and it is the leading cause of death. Despite this, heart and blood vessel (cardiovascular system) manifestations of Fabry disease remain poorly characterised, and it remains unclear which patients benefit from therapy, or when therapy should be initiated. Migalastat is increasingly used to treat fabry disease however the impact of Migalastat on the cardiovascular system is poorly understood. Detailed assessment of the impact of Migalastat on heart and blood vessel structure and function is urgently needed. This observational study will use state of the art, non-invasive investigations to provide greater understanding of the cardiovascular manifestations of Fabry disease and the effects of Migalastat. It will provide insight into which patients respond more effectively to Migalastat, which in turn will facilitate personalisation of therapy, optimisation of the timing of therapy initiation and more cost-effective care.

Conditions

Interventions

DRUG

Migalastat

Participants will be starting Migalastat as part of clinical care. Their initiation onto Migalastat is not determined by this study or it's protocol. Interventions as part of the study will be limited to the study investigations.

Sponsors & Collaborators

  • Northern Care Alliance NHS Foundation Trust

    collaborator OTHER

  • Manchester University NHS Foundation Trust

    lead OTHER_GOV

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-05-16
Primary Completion
2023-12-31
Completion
2023-12-31

Countries

  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03949920 on ClinicalTrials.gov