A Study of Migalastat in Fabry Disease

Fabry Disease Clinical Trial

— GALAFAB  

Official title:

A Prospective Observational Study Investigating the Impact of Migalastat on Cardiovascular Structure and Function in Fabry Disease

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03949920
• Other study ID #: B00544
• Secondary ID: 19/NW/0099
• Status: Active, not recruiting
• Start date: May 16, 2019
• Est. completion date: December 31, 2023

Study information

• Verified date: May 2023
• Source: Manchester University NHS Foundation Trust
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 21
• Est. completion date: December 31, 2023
• Est. primary completion date: December 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

Contraindication to cardiac MRI

Related Conditions & MeSH terms

• Fabry Disease

Intervention

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.

Locations

Country	Name	City	State
United Kingdom	Manchester University Foundation Trust	Manchester
United Kingdom	Salford Royal NHS Foundation Trust	Manchester

Sponsors (2)

Lead Sponsor	Collaborator
Manchester University NHS Foundation Trust	Northern Care Alliance NHS Foundation Trust

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Indexed Left Ventricular Mass (grams/m2)	LV mass, indexed to body surface area, assessed using cardiac MRI.	12 months
Secondary	Change in BSA-indexed LV volumes measured using cardiac MRI, from baseline to 12 months (mls/m2)		12 months
Secondary	Change LV ejection fraction, measured using cardiac MRI (%)		12 months
Secondary	Change in myocardial extracellular volume, measured using cardiac MRI (%)		12 months
Secondary	Change in myocardial tissue T1 and T2 times measured using cardiac MRI (ms)		12 months
Secondary	Change in pulmonary artery systolic pressure, measured using echocardiography (mmHg)		12 months
Secondary	Change in myocardial PCr/ATP ratio, measured using cardiac MRI spectroscopy (ratio)		12 months
Secondary	Change in the number of extra heart beats in a 24 hour period, measured using ambulatory heart monitoring. (number)		12 months
Secondary	Change in exercise capacity - Six minute walk test (meters)		12 months
Secondary	20. Change in health status (quality of life), measured using change in SF-36 score. (score)		12 months
Secondary	Change in Lyso-GB3 blood test levels (nmol/L)		12 months
Secondary	Change in Troponin blood test levels (ng/L)		12 months
Secondary	Change in NT pro BNP blood test levels (pg/L)		12 months