Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Information source: National Center for Research Resources (NCRR)
Information obtained from ClinicalTrials.gov on October 19, 2009
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher's Disease
Intervention: glucocerebrosidase (Drug)
Phase: Phase 2
Status: Recruiting
Sponsored by: National Center for Research Resources (NCRR) Official(s) and/or principal investigator(s):
John Barranger, Study Chair, Affiliation: University of Pittsburgh
Summary
OBJECTIVES:
I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with
Gaucher disease.
Clinical Details
Study design: Treatment
Detailed 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.
Eligibility
Minimum age: 18 Years.
Maximum age: 65 Years.
Gender(s): Both.
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
Locations and Contacts
University of Pittsburgh, Pittsburgh, Pennsylvania 15260, United States; Recruiting
John Barranger, Phone: 412-624-4623
Additional Information
Starting date: November 1999
Last updated: June 23, 2005
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