Back to resultsPhase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Primary Purpose
Gaucher's Disease
Status
Unknown status
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
glucocerebrosidase
Sponsored by
About this trial
This is an interventional treatment trial for Gaucher's Disease focused on measuring Gaucher's disease, inborn errors of metabolism, rare disease, sphingolipidoses
Eligibility 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
Sites / Locations
- University of PittsburghRecruiting
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004293
First Posted
October 18, 1999
Last Updated
June 23, 2005
Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Pittsburgh
1. Study Identification
Unique Protocol Identification Number
NCT00004293
Brief Title
Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Study Type
Interventional
2. Study Status
Record Verification Date
December 2003
Overall Recruitment Status
Unknown status
Study Start Date
November 1999 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Pittsburgh
4. Oversight
5. Study Description
Brief Summary
OBJECTIVES:
I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher's Disease
Keywords
Gaucher's disease, inborn errors of metabolism, rare disease, sphingolipidoses
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Enrollment
24 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
glucocerebrosidase
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John Barranger
Organizational Affiliation
University of Pittsburgh
Official's Role
Study Chair
Facility Information:
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15260
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
John Barranger
Phone
412-624-4623
12. IPD Sharing Statement
Learn more about this trial
Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
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