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Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00004293
Recruitment Status : Unknown
Verified December 2003 by National Center for Research Resources (NCRR).
Recruitment status was:  Recruiting
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
Sponsor:
Collaborator:
University of Pittsburgh
Information provided by:
National Center for Research Resources (NCRR)

Study Description
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Brief Summary:

OBJECTIVES:

I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.


Condition or disease Intervention/treatment Phase
Gaucher's Disease Drug: glucocerebrosidase 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
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Study Type : Interventional  (Clinical Trial)
Enrollment : 24 participants
Primary Purpose: Treatment
Study Start Date : November 1999

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Arms and Interventions
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Outcome Measures
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Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004293


Locations
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United States, Pennsylvania
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15260
Contact: John Barranger    412-624-4623      
Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of Pittsburgh
Investigators
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Study Chair: John Barranger University of Pittsburgh
More Information
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ClinicalTrials.gov Identifier: NCT00004293    
Other Study ID Numbers: 199/11725
UPITTS-M1230
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: December 2003
Keywords provided by National Center for Research Resources (NCRR):
Gaucher's disease
inborn errors of metabolism
rare disease
sphingolipidoses
Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders