NCT05054387

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

Summary

This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.

Detailed description

Study participation for each patient will be total of 54 weeks which will include 4 weeks of screening, 48 weeks of treatment period and 2 weeks of post study treatment observation

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Agalsidase beta	Powder for concentration into a solution Intravenous (IV) infusion

Timeline

• Start date: 2021-10-13
• Primary completion: 2023-03-09
• Completion: 2023-03-09
• First posted: 2021-09-23
• Last updated: 2025-09-11

Locations

6 sites across 1 country: China