CLI-06657AA1-04 Open Label Extension Study to Evaluate Alfa (PRX-102)
Research type
Research Study
Full title
Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
IRAS ID
249199
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Chiesi Farmaceutici S.p.A
Eudract number
2018-001148-67
ISRCTN Number
ISRCTN12345789
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
NA, NA
Duration of Study in the UK
4 years, 10 months, 15 days
Research summary
The purpose of this research study is to test the experimental drug called pegunigalsidase alfa (PRX-102) as an Enzyme Replacement Therapy for Fabry disease. Participants are being asked to be in this study because they have Fabry disease and have successfully completed a previous research study for pegunigalsidase alfa or in some instances, agalsidase beta (Fabrazyme®). Participants are being offered to participate in the study to see if pegunigalsidase alfa is safe and effective for treatment of Fabry disease over time. Around 110 patients in several countries will take part in this study. Duration of the study is no less than 24 months and up to 48 months or until pegunigalsidase alfa is commercially available to the patient at the discretion of the Sponsor.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
18/EE/0241
Date of REC Opinion
25 Sep 2018
REC opinion
Further Information Favourable Opinion