PRX-102 compared to Agalsidase Beta in Patients with Fabry Disease
Research type
Research Study
Full title
A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta
IRAS ID
210027
Contact name
Louisa Vass
Contact email
Sponsor organisation
Protalix Ltd.
Eudract number
2016-000378-38
Duration of Study in the UK
2 years, 7 months, 20 days
Research summary
The purpose of this research study is to test the experimental drug called PRX-102 as an enzyme replacement therapy for Fabry disease. Fabry disease is a rare, inherited disease caused by the deficiency of a certain enzyme which results in reduced kidney function. Patients who are currently being treated with a standard enzyme replacement therapy called agalsidase beta (also known as Fabrazyme) will be invited to participate in the study to see if the PRX-102 will improve or decrease the effects of Fabry disease on their kidney function. Around 78 patients, in 20 study centres globally will take part in this study.
This study will look at the safety of PRX-102, its effects on Fabry disease, and how well it is tolerated by participants. As part of the study design, some participants will be treated with PRX-102 and some will continue to be treated with Fabrazyme. Participants will have a 2:1 chance of being assigned to receive PRX-102. This means that twice as many participants will be treated with PRX-102 as with Fabrazyme. Neither the participant nor the study doctor will know which treatment the participant has been assigned to.
Participation will last about 2 years and will include study visits every 2 weeks for a total of 53 intravenous (through the vein) infusions (participants will receive intravenous infusions of PRX-102 1 mg/kg or agalsidase beta 1 mg/kg). At each visit, the participant will have various tests and procedures (safety assessments) and will be closely monitored while they are on study to see whether there is an improvement in the effects of Fabry disease on their kidney function. At the end of the 2-year treatment period, eligible participants may be offered to take part in an additional open-label extension study where all participants will be treated with the enzyme PRX-102.
REC name
London - Westminster Research Ethics Committee
REC reference
16/LO/1466
Date of REC Opinion
7 Sep 2016
REC opinion
Favourable Opinion