MODERN study
Research type
Research Study
Full title
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients MODERN
IRAS ID
347172
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Chiesi USA, Inc
Duration of Study in the UK
5 years, 1 months, 11 days
Research summary
Pegunigalsidase alfa was recently approved by the FDA (May 2023), EMA (May 2023) and MHRA (August 2023) for treatment of Fabry disease. Following approval, there is a need to generate realworld evidence to further understand its long-term impact in specific patient subgroups, as well as to characterize its tolerability, safety, and effectiveness during routine clinical care. Analysing the real-world impact/use of patients treated with pegunigalsidase alfa will enrich knowledge on FD management with this recently approved product. Three patient cohorts were identified as representing patient populations likely to contribute the most valuable data: treatment naïve patients, patients with cardiac impairment due to Fabry disease, and patients from the open label extension study (OLE) CLI-06657AA1-04 (Previously PB-102-F60) who are transitioning to commercial product. Data will be obtained over four years of follow up through routine clinical monitoring from sites across multiple countries in Europe and North America.
REC name
Yorkshire & The Humber - Bradford Leeds Research Ethics Committee
REC reference
24/YH/0251
Date of REC Opinion
8 Nov 2024
REC opinion
Favourable Opinion