NCT02795676

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 78 (actual)
• Sponsor: Protalix · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients who had been treated for approximately 1 year with agalsidase beta and who had been on a stable dose of that product for at least 6 months were randomized in a 2:1 ratio to either switch to PRX-102 or to continue treatment with agalsidase beta. Both treatments were delivered by intravenous infusions every two weeks, at a dosage of 1 mg/kg.

Detailed description

This was a randomized, double-blind, active control study examining the safety and efficacy of pegunigalsidase alfa (PRX-102) in Fabry disease patients with impaired renal function. Participants had to have been taking the licensed ERT drug agalsidase beta (Fabrazyme®) for at least 1 year prior to study entry, and to have been on a stable dose of that product for at least the last 6 months. Since the disease expresses itself differently in males and females, gender could have an impact on the therapeutic effect; thus, there was additionally a requirement that no more than 50% of the patients could be female. Following screening, eligible patients were randomized in a 2:1 ratio to either switch to PRX-102 or continue treatment with agalsidase beta, with randomization stratified according to whether the urine protein-to-creatinine ratio (UPCR), a measure of kidney function, was above or below a specified threshold. Both products were administered as an intravenous infusion every 2 weeks, at a dosage of 1 mg/kg, for up to 24 months. Both patients and study staff were blinded as to which treatment was being given. Patients who completed the study were invited to continue in a long-term open-label extension study, PB-102-F60, in which all participants would receive PRX-102 1 mg/kg every 2 weeks.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	PRX-102 (pegunigalsidase alfa)	PRX-102 1 mg/kg every 2 weeks
BIOLOGICAL	agalsidase beta	agalsidase beta 1 mg/kg every 2 weeks

Timeline

• Start date: 2016-06-01
• Primary completion: 2021-10-01
• Completion: 2022-07-01
• First posted: 2016-06-10
• Last updated: 2023-09-13
• Results posted: 2023-08-02

Locations

29 sites across 12 countries: United States, Czechia, Finland, France, Hungary, Italy, Netherlands, Norway, Slovenia, Spain, Switzerland, United Kingdom