Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
NCT ID: NCT00004293
Last Updated: 2005-06-24
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2
24 participants
INTERVENTIONAL
1999-11-30
Brief Summary
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I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.
Detailed Description
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Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.
Conditions
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Study Design
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TREATMENT
Interventions
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glucocerebrosidase
Eligibility Criteria
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Inclusion 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
18 Years
65 Years
ALL
No
Sponsors
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University of Pittsburgh
OTHER
National Center for Research Resources (NCRR)
NIH
Principal Investigators
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John Barranger
Role: STUDY_CHAIR
University of Pittsburgh
Locations
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University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Countries
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Facility Contacts
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John Barranger
Role: primary
Other Identifiers
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UPITTS-M1230
Identifier Type: -
Identifier Source: secondary_id
199/11725
Identifier Type: -
Identifier Source: org_study_id