Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Status:
Approved for marketing
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
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 treatment protocol is to observe the safety of
velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive
(newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy
(ERT) imiglucerase.