NCT00701415

A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms

A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (Agalsidase Beta) in Treatment-Naïve Male Pediatric Patients With Fabry Disease Without Severe Symptoms

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 5 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without severe symptoms. Participants were to be treated for 5 years.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	Agalsidase beta	Powder for concentrate for solution for infusion 1.0 mg/kg/4 weeks
BIOLOGICAL	Agalsidase beta	Powder for concentrate for solution for infusion 0.5 mg/kg/2 weeks

Timeline

• Start date: 2008-09-01
• Primary completion: 2015-06-01
• Completion: 2015-06-01
• First posted: 2008-06-19
• Last updated: 2016-06-29
• Results posted: 2016-06-29

Locations

12 sites across 9 countries: United States, Argentina, Brazil, Canada, Czechia, Netherlands, Norway, Poland, United Kingdom