NCT00635427

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 95 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.

Detailed description

Type 1 Gaucher disease, the most common form,accounts for more than 90% of all cases and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (GA-GCB,velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. GA-GCB contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the long-term safety of GA-GCB in men, women, and children with Type 1 Gaucher disease.

Conditions

• Gaucher Disease, Type 1

Interventions

Type	Name	Description
BIOLOGICAL	VPRIV®	Intravenous infusion, every other week (EOW)

Timeline

• Start date: 2008-03-13
• Primary completion: 2012-12-28
• Completion: 2012-12-28
• First posted: 2008-03-13
• Last updated: 2021-06-10
• Results posted: 2014-01-28

Locations

21 sites across 11 countries: United States, Argentina, India, Israel, Paraguay, Poland, Russia, South Korea, Spain, Tunisia, United Kingdom