Prospective Observational Registry of Patients with Fabry Disease
Research type
Research Study
Full title
A Prospective, Observational Registry of Patients with Fabry Disease
IRAS ID
242638
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Amicus Therpeutics UK Ltd.
Clinicaltrials.gov Identifier
EUPAS20599, European Union Post- authorisation Study (EU PAS) register number; EU/1/15/1082/001, Product Reference; EMEA/H/C/004059/MEA/001, Procedure Number
Duration of Study in the UK
9 years, 0 months, 1 days
Research summary
Fabry disease is a rare, progressive disease. The disease is caused by mutation in the GLA gene that results in the deficiency of the lysosomal enzyme (alpha Galactosidase A.) Available treatments for Fabry disease are intravenous enzyme replacement therapy (ERT), such as agalsidase alfa or agalsidase beta, and migalastat, an oral pharmacological chaperone.
This registry is designed to assess the effects of Migalastat treatment in patients with Fabry's disease. The registry will evaluate the long-term safety, effectiveness, and quality of life (QOL) in patients with Fabry disease, with a main focus on migalastat, a medicine with a novel mechanism of action.
All visits will be scheduled and conducted according to the clinical site’s standard of care. Standard of care is defined as the customary clinical treatment/practice process that a clinician chooses according to their clinical judgement for a Fabry disease patient. There are no registry required visits, tests or clinical assessments.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
18/EE/0144
Date of REC Opinion
23 Aug 2018
REC opinion
Further Information Favourable Opinion