NCT01363492

Safety Study of Replagal® Therapy in Children With Fabry Disease

An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 7 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).

Detailed description

In 2008, a change in the agalsidase alfa drug substance manufacturing process was made. There are no changes to the drug product formulation, manufacturing site, manufacturing process, or container closure. An agalsidase alfa bioreactor manufacturing process (agalAF1) utilizing animal component-free media replaced the previous roller bottle (RB) process. This study will evaluate the safety of Replagal AF, manufactured using the new bioreactor process at a dose of 0.2 mg/kg infused IV over 40 minutes, every other week (EOW) in children with Fabry disease who are 7 years to less than 18 years of age and who are naive to ERT.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	Replagal (agalsidase alfa)	0.2 mg/kg administered over 40 minutes every other week (EOW)

Timeline

• Start date: 2011-05-12
• Primary completion: 2013-04-17
• Completion: 2013-04-17
• First posted: 2011-06-01
• Last updated: 2021-06-09
• Results posted: 2014-05-20

Locations

5 sites across 1 country: United States