Expired Study
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Pittsburgh, Pennsylvania 15260


Purpose:

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


Study summary:

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


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


NCT ID:

NCT00004293


Primary Contact:

Study Chair
John Barranger
University of Pittsburgh


Backup Contact:

N/A


Location Contact:

Pittsburgh, Pennsylvania 15260
United States

John Barranger
Phone: 412-624-4623

Site Status: Recruiting


Data Source: ClinicalTrials.gov

Date Processed: September 23, 2017

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