NCT00553631

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this non-inferiority study is to evaluate the efficacy and safety of GA-GCB (velaglucerase alfa) administered every other week in comparison to imiglucerase in treatment naive 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 (velaglucerase alfa) 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 efficacy and safety of GA-GCB (velaglucerase alfa) in comparison to imiglucerase in men, women, and children with Type 1 Gaucher disease.

Conditions

• Gaucher Disease, Type 1

Interventions

Type	Name	Description
BIOLOGICAL	velaglucerase alfa	IV infusion, 60 U/kg every other week for 9 months
BIOLOGICAL	imiglucerase	IV infusion, 60 U/kg every other week for 9 months

Timeline

• Start date: 2008-01-29
• Primary completion: 2009-05-05
• Completion: 2009-05-05
• First posted: 2007-11-05
• Last updated: 2021-06-08
• Results posted: 2011-01-04

Locations

11 sites across 9 countries: United States, Argentina, India, Israel, Paraguay, Russia, Spain, Tunisia, United Kingdom