Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

National Center for Research Resources (NCRR)

Status and phase: Unknown; Phase 2

Conditions: Gaucher's Disease

Treatments: Drug: glucocerebrosidase

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT00004293

199/11725

UPITTS-M1230

Details and patient eligibility

About

OBJECTIVES:

I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.

Full description

PROTOCOL OUTLINE:

Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Gaucher disease with glucocerebrosidase deficiency confirmed by enzymatic or molecular assay At least 3 organ systems affected, based on the following criteria: Anemia Thrombocytopenia Organomegaly Bone deterioration on radiograph Pulmonary compromise Symptoms compromise daily activities or risk longevity No neurologic disease