Clinical Trials Logo

Gaucher Disease clinical trials

View clinical trials related to Gaucher Disease.

Filter by:
  • Approved for marketing  
  • Page 1

NCT ID: NCT00954460 Approved for marketing - Clinical trials for Gaucher Disease, Type 1

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Start date: n/a
Phase:
Study type: Expanded Access

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.