You are viewing an expired study

Pittsburgh, Pennsylvania 15260

  • Gaucher's Disease


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.


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



Primary Contact:

Study Chair
John Barranger
University of Pittsburgh

Backup Contact:


Location Contact:

Pittsburgh, Pennsylvania 15260
United States

John Barranger
Phone: 412-624-4623

Site Status: Recruiting

Data Source:

Date Processed: March 30, 2020

Modifications to this listing: Only selected fields are shown, please use the link below to view all information about this clinical trial.

Click to view Full Listing

This study is not currently recruiting Study Participants on The form below is not enabled.