Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
Status:
Completed
Trial end date:
2014-10-08
Target enrollment:
Participant gender:
Summary
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB)
that leads to progressive accumulation of glucocerebroside within macrophages and subsequent
tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1
Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1
Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher
disease present with acute neurological deterioration, which leads to early death. Those with
Type 3 disease typically display a more sub-acute neurological course, with later onset and
slower progression.
The primary objective of this study is to evaluate the long-term safety of every other week
(EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed
study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa.
Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type
1 Gaucher disease.