Ext Study AT1001-042- Effects of AT1001 in Patients with Fabry Disease
Research type
Research Study
Full title
An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease
IRAS ID
169064
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Amicus Therapeutics, Inc.
Eudract number
2014-002701-38
Clinicaltrials.gov Identifier
Duration of Study in the UK
6 years, 1 months, 1 days
Research summary
This is a phase 3, open label, non-comparative, extension study to evaluate the long term safety, efficacy, action and effects of the investigational drug migalastat hydrochloride (HCl) in the treatment of subjects with Fabry disease who have completed treatment in the previous study of migalastat HCl. Subjects will be recruited from previous studies, AT1001-041 and AT1001-012.
Patients in the UK will enter this study with their Baseline Visit 1 being performed at the same time of their final visit in the previous study. The anticipated participation period for each patient is 5 years (60 months). Patients will undertake baseline measurements on the same day as the final treatment visit for the previous study, followed by a clinic visit every 6 months over a 5 year period. Patients will be required to orally take one 150mg capsule of the study drug, every other day. Dummy capsules will also be provided to take on alternate days to act as a reminder. Patients will receive a telephone call or other suitable communication at half way intervals between visits to monitor adverse events and dosing compliance. An end of study visit will be arranged approximately 30 days after last treatment.This is a multicentre international study with approximately 50 study sites.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
14/EM/1308
Date of REC Opinion
28 Jan 2015
REC opinion
Further Information Favourable Opinion