Fabry study with Fabrazyme and Replagal
Research type
Research Study
Full title
A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease.
IRAS ID
266648
Contact name
John Salcedo
Contact email
Sponsor organisation
Sanofi Aventis Groupe (SAG)
Eudract number
2019-000064-21
Duration of Study in the UK
2 years, 9 months, 1 days
Research summary
The purpose of the study is to assess the ability of the Fabrazyme (agalsidase beta) to reduce glycosphingolipid deposition in blood, kidney and skin of the participants after switching from comparator drug Replagal (agalsidase alpha) to Fabrazyme against those who continue taking Replagal. Glycosphingolipid is an accumulated fatty substance in various tissues of the body of Fabry disease patients due to deficiency of an enzyme named alpha-galactosidase A. This study will also assess the changes of kidney and heart function which are important parameters to measure disease status. Finally, changes in disease severity and Fabry disease symptoms will be measured.
The study will last around 61 weeks and is planned to include approximately 28 participants worldwide. The study will include only male Fabry patients who have been prescribed and receiving Replagal for their disease management.
REC name
London - Hampstead Research Ethics Committee
REC reference
19/LO/1864
Date of REC Opinion
20 Dec 2019
REC opinion
Favourable Opinion