A Study of Migalastat in Fabry Disease (GALAFAB)
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|ClinicalTrials.gov Identifier: NCT03949920|
Recruitment Status : Not yet recruiting
First Posted : May 14, 2019
Last Update Posted : May 14, 2019
|Condition or disease||Intervention/treatment|
|Fabry Disease||Drug: Migalastat|
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.
|Study Type :||Observational|
|Estimated Enrollment :||21 participants|
|Official Title:||A Prospective Observational Study Investigating the Impact of Migalastat on Cardiovascular Structure and Function in Fabry Disease|
|Estimated Study Start Date :||June 1, 2019|
|Estimated Primary Completion Date :||December 1, 2021|
|Estimated Study Completion Date :||February 1, 2022|
- 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.
- Indexed Left Ventricular Mass (grams/m2) [ Time Frame: 12 months ]LV mass, indexed to body surface area, assessed using cardiac MRI.
- Change in BSA-indexed LV volumes measured using cardiac MRI, from baseline to 12 months (mls/m2) [ Time Frame: 12 months ]
- Change LV ejection fraction, measured using cardiac MRI (%) [ Time Frame: 12 months ]
- Change in myocardial extracellular volume, measured using cardiac MRI (%) [ Time Frame: 12 months ]
- Change in myocardial tissue T1 and T2 times measured using cardiac MRI (ms) [ Time Frame: 12 months ]
- Change in pulmonary artery systolic pressure, measured using echocardiography (mmHg) [ Time Frame: 12 months ]
- Change in myocardial PCr/ATP ratio, measured using cardiac MRI spectroscopy (ratio) [ Time Frame: 12 months ]
- Change in the number of extra heart beats in a 24 hour period, measured using ambulatory heart monitoring. (number) [ Time Frame: 12 months ]
- Change in exercise capacity - Six minute walk test (meters) [ Time Frame: 12 months ]
- 20. Change in health status (quality of life), measured using change in SF-36 score. (score) [ Time Frame: 12 months ]
- Change in Lyso-GB3 blood test levels (nmol/L) [ Time Frame: 12 months ]
- Change in Troponin blood test levels (ng/L) [ Time Frame: 12 months ]
- Change in NT pro BNP blood test levels (pg/L) [ Time Frame: 12 months ]
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03949920
|Contact: Chris Miller, BSc MBChB MRCP PhD||0161 291 4075 ext firstname.lastname@example.org|
|Contact: Chris Orsborne, BSc MBChB MRCP||0161 291 4075 ext email@example.com|
|Manchester University Foundation Trust||Not yet recruiting|
|Manchester, United Kingdom, M139LT|
|Contact: Chris Miller, BSc, MBChB, PhD, MRCP 0161 291 4075 ext 4075 Christopher Miller <firstname.lastname@example.org>|
|Contact: Chris Orsborne, BSc, MBChB, MRCP 0161 291 4075 ext 4075 email@example.com|
|Principal Investigator: Chris Orsborne, BSc, MBChB, MRCP|
|Salford Royal NHS Foundation Trust||Not yet recruiting|
|Manchester, United Kingdom, M6 8HD|
|Contact: Ana Jovanovic 0161 206 1080 ext 61080 Ana.Jovanovic@srft.nhs.uk|
|Contact: Karen Wynne 0161 206 1080 ext 60180 firstname.lastname@example.org|