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Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Sponsor
National Center for Research Resources (NCRR) (NIH)
Overall Status
Unknown status
CT.gov ID
NCT00004293
Collaborator
University of Pittsburgh (Other)
24
Enrollment
1
Location

Study Details

Study Description

Brief Summary

OBJECTIVES:
  1. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.
Condition or Disease Intervention/Treatment Phase
Phase 2

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.

Study Design

Study Type:
Interventional
Primary Purpose:
Treatment
Study Start Date :
Nov 1, 1999

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 65 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Pittsburgh Pittsburgh Pennsylvania United States 15260

    Sponsors and Collaborators

    • National Center for Research Resources (NCRR)
    • University of Pittsburgh

    Investigators

    • Study Chair: John Barranger, University of Pittsburgh

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    , ,
    ClinicalTrials.gov Identifier:
    NCT00004293
    Other Study ID Numbers:
    • 199/11725
    • UPITTS-M1230
    First Posted:
    Oct 19, 1999
    Last Update Posted:
    Jun 24, 2005
    Last Verified:
    Dec 1, 2003
    Keywords provided by , ,
    Gaucher's disease
    inborn errors of metabolism
    rare disease
    sphingolipidoses
    Additional relevant MeSH terms:
    Gaucher Disease

    Study Results

    No Results Posted as of Jun 24, 2005