NCT03566017

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 97 (actual)
• Sponsor: Chiesi Farmaceutici S.p.A. · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The objective of CLI-06657AA1-04 (formerly PB-102-F60) was to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who had successfully completed studies PB-102-F20, PB-102-F30, or at least 48 months in study PB-102-F03.

Detailed description

This was an open-label study. Participants were enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment was until pegunigalsidase alfa was commercially available to the participant, or at the discretion of the Sponsor. Efficacy and safety analyses were summarized using descriptive statistics for continuous variables and counts and percentages for categorical variables. Data from the parent studies were also included in the analyses.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	pegunigalsidase alfa	Recombinant human alpha galactosidase A

Timeline

• Start date: 2018-09-16
• Primary completion: 2025-01-21
• Completion: 2025-01-21
• First posted: 2018-06-21
• Last updated: 2026-03-24
• Results posted: 2026-03-24

Locations

30 sites across 13 countries: United States, Australia, Canada, Czechia, Finland, France, Hungary, Italy, Netherlands, Norway, Slovenia, Spain, United Kingdom

Regulatory

• FDA-regulated drug study