CLI-06657AA1-03 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) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks in Patients with Fabry Disease
IRAS ID
264276
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Chiesi Farmaceutici S.p.A
Eudract number
2018-001947-30
ISRCTN Number
ISRCTN12345789
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
3 years, 6 months, 1 days
Research summary
This study is an extension study to F50 and will be an open-label, multicentre study of 2 mg/kg of pegunigalsidase alfa intravenous infusion every 4 weeks (±3 days) to evaluate the safety and efficacy of pegunigalsidase alfa in adult Fabry patients (≥18 years of age).
The duration of treatment will be up to 36 months or until pegunigalsidase alfa is available to the patient or at the discretion of the Sponsor.
In the case of clear clinical deterioration, the treatment may be changed to 1.0 mg/kg every 2 weeks at the Investigator’s discretion and discussion with the Medical Monitor.
All disease parameters that were evaluated during study PB-102-F50 will continue to be assessed in this extension protocol (study PB-102-F51).
The extension study may provide additional, long-term information on safety, and efficacy in patients treated with pegunigalsidase alfa under a regimen of 2 mg/kg every 4 weeks.REC name
West of Scotland REC 1
REC reference
19/WS/0064
Date of REC Opinion
27 May 2019
REC opinion
Further Information Favourable Opinion