Relation between enzyme replacement therapy and progression of brain lesions in Fabry disease

Not Applicable

Completed

• Conditions: Nutritional, Metabolic, Endocrine; Fabry disease

• Registration Number: ISRCTN14072182
• Lead Sponsor: niversity of Manchester

Brief Summary

2018 results in: https://doi.org/10.1212/WNL.0000000000006316 (added 14/06/2019)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-02-10
• Study Completion: 2016-12-31
• Last Update Posted: 15 July 2019

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 149

Inclusion Criteria

1. Confirmed diagnosis of Fabry disease
2. Age at least 18 years old
3. Being followed up at Salford Royal NHS Foundation Trust (UK)
4. Registered in Fabry disease registry
5. At least two serial MRI brain scans (1 year apart)

Exclusion Criteria

1. New patient
2. No serial MRI scans
3. MRI scans of insufficient quality for analysis

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Prevalence and burden of white matter lesions over time: MRI scans at baseline and at 2 years will be compared for evidence of white-matter lesions using a visual severity rating scale

Secondary Outcome Measures

Name	Time	Method
Progression of white matter lesions: MRI scans at baseline and at 2 years will be compared for evidence of white-matter lesions using a visual severity rating scale