PB-102-F30-Open Label Study of PRX-102 in patients with Fabry Disease
Research type
Research Study
Full title
An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)
IRAS ID
220369
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Protalix Ltd.
Eudract number
2016-001318-11
Duration of Study in the UK
1 years, 6 months, 25 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 alfa (also known as REPLAGAL®) will be invited to participate in the study. This is an open label switch over study to assess the safety and effectiveness of PRX-102 for the treatment of Fabry disease. Patients treated with agalsidase alfa for at least 2 years and on a stable dose for at least 6 months, will be screened and evaluated over 3 months while continuing on agalsidase alfa. Following the screening period, the patient will be enrolled and switched from their agalsidase alfa treatment to receive intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25% of treated patients will be female.22 participants will take part in the study globally, with a target of 8 participants to be recruited in the UK amoungst 3 research sites.
At the time of enrolment, premedication, if used for the agalsidase alfa infusions before enrolment, will be continued through the first infusion with PRX-102 and then will be gradually tapered at the investigator’s discretion during the first 2 months. The first infusions of PRX-102 will be administered under controlled conditions at the investigation site. In this study, there is the option that patient can receive their PRX-102 infusions at a home care setup once the investigator and Sponsor Medical Director agree that it is safe to do so. Home care set up would only be set up if this is a feasible and safe option.
REC name
London - Hampstead Research Ethics Committee
REC reference
17/LO/0040
Date of REC Opinion
24 Mar 2017
REC opinion
Further Information Favourable Opinion