Chiesi_PEOPLE
Research type
Research Study
Full title
Fabry Patient’s Experience Of PegunigaLsidasE Alfa Monthly Infusion – PEOPLE Study (CLI-06657AA1-05)
IRAS ID
302792
Contact name
Derralynn Arlene Hughes
Contact email
Sponsor organisation
CHIESI FARMACEUTICI S.p.A.
Duration of Study in the UK
0 years, 4 months, 1 days
Research summary
The purpose of this qualitative study, conducted through a 60-minute interview, is to further understand the experience of patients affected by Fabry’s disease (FD) who have participated in the PB-102-F50 clinical trial and are participating in its extension study, PB-102-F51.
Fabry disease (FD) is a rare inherited lysosomal storage disorder caused by deficiency of α-galactosidase A (α-GAL). This deficiency results in the accumulation of globotriaosylceramide in lysosomes which is associated with cellular dysfunction. Over time, this dysfunction leads to progressive and irreversible organ damage.
The study will involve participants over 18 years with Fabry Disease who are participating in the PB-102-F51 extension study to also participate in this qualitative research. The objective of this study is to describe the patients’ experience of Fabry Disease symptoms when treated with Pegunigalsidase Alfa (PRX-102) every 4 weeks for over 2 years. This study plans to interview up to 29 patients with Fabry Disease, including patients from several study centres in Europe and North America. This study is organized and paid for by Chiesi Farmaceutici S.p.A, Via Palermo 26/A, 43122 Parma (Italy).Apart from any direct benefit to you, the participation of the participant is likely to help us understand patients’ experience of Fabry Disease symptoms and add knowledge about the impact of infusions of PRX-102 on quality of life in Fabry Disease patients.REC name
North West - Haydock Research Ethics Committee
REC reference
21/NW/0364
Date of REC Opinion
17 Feb 2022
REC opinion
Further Information Favourable Opinion