NCT03614234

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Chiesi Farmaceutici S.p.A. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Detailed description

This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	pegunigalsidase alfa	Recombinant human alpha galactosidase A

Timeline

• Start date: 2018-11-13
• Primary completion: 2026-04-30
• Completion: 2026-06-30
• First posted: 2018-08-03
• Last updated: 2026-01-21

Locations

14 sites across 7 countries: United States, Belgium, Czechia, Denmark, Italy, Norway, United Kingdom

Regulatory

• FDA-regulated drug study