Fabry SRT Mono Extension
Research type
Research Study
Full title
An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed with Fabry Disease
IRAS ID
179349
Contact name
Patrick Deegan
Contact email
Sponsor organisation
Genzyme Corporation
Eudract number
2014-004995-49
Duration of Study in the UK
3 years, 2 months, 30 days
Research summary
An open-label, multicenter, multinational extension study to evaluate the long-term safety and efficacy of study medicine in adult male patients diagnosed with Fabry disease.
The purpose of this study is to assess the long-term safety and effectiveness of the study medicine at 15 mg dose taken orally once daily for 30 months in adult male patients diagnosed with Fabry disease and completed the ACT13739 study.
The total duration of your participation in the study will be 31 months. This study comprises of:
•a 30 month treatment period
•Post-treatment follow up: 1 month (± 7 days). Patients who start commercial ERT within the 1-month follow up period will not be contacted for follow up.Primary objective: To assess the long-term safety of GZ/SAR402671 in adult male patients with Fabry disease who previously completed study ACT13739.
REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
15/EE/0210
Date of REC Opinion
14 Oct 2015
REC opinion
Further Information Favourable Opinion