NCT03018730

Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

An Open Label Study of the Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Protalix · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This is an open label switch over study to assess the safety and efficacy of PRX-102 (pegunigalsidase alfa). Patients treated with agalsidase alfa for at least 2 years and on a stable dose (\>80% labelled dose/kg) for at least 6 months. Patients will be screened and evaluated over 3 months while continuing on agalsidase alfa. Following the screening period, the patient will be enrolled and switched from their agalsidase alfa treatment to receive intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25% of treated patients will be female.

Detailed description

Dosage and administration details: pegunigalsidase alfa individual dose for each patient was prepared according to the patient's weight. Pegunigalsidase alfa administrated at 1 mg/kg, intravenously over 3 hours, every 2 weeks. After the first 2 months of treatment with pegunigalsidase alfa, infusion time may be reduced gradually to 1.5 hours pending patient tolerability.

Conditions

• Fabry Disease

Interventions

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

Timeline

• Start date: 2017-05-17
• Primary completion: 2019-12-17
• Completion: 2020-01-09
• First posted: 2017-01-12
• Last updated: 2023-09-13
• Results posted: 2022-06-29

Locations

9 sites across 7 countries: Australia, Canada, Czechia, Netherlands, Norway, Slovenia, United Kingdom

Regulatory

• FDA-regulated drug study