A Study of Migalastat in Fabry Disease (GALAFAB)

  • STATUS
    Recruiting
  • End date
    Dec 31, 2023
  • participants needed
    21
  • sponsor
    Manchester University NHS Foundation Trust
Updated on 21 March 2022
migalastat

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.

Description

This is a prospective longitudinal observational study of patients starting Migalastat as part of routine care. Participants will be recruited from outpatient clinics and have a number of investigations before starting therapy and after twelve months of therapy.

Investigations will include a detailed assessment of symptoms and clinical features, blood tests, echocardiography, detailed cardiac MRI scans, heart rhythm monitoring and exercise capacity assessment.

Parameters will be assessed at baseline and at twelve months.

Details
Condition Fabry Disease
Treatment migalastat
Clinical Study IdentifierNCT03949920
SponsorManchester University NHS Foundation Trust
Last Modified on21 March 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Confirmed Fabry disease Aged 16 or over Beginning clinical treatment with
Migalastat

Exclusion Criteria

Contraindication to cardiac MRI
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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