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Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified December 2003 by National Center for Research Resources (NCRR).
Recruitment status was:  Recruiting
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004293
First Posted: October 19, 1999
Last Update Posted: December 9, 2005
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.
Collaborator:
University of Pittsburgh
Information provided by:
National Center for Research Resources (NCRR)
  Purpose

OBJECTIVES:

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


Condition Intervention Phase
Gaucher's Disease Drug: glucocerebrosidase Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by National Center for Research Resources (NCRR):

Estimated Enrollment: 24
Study Start Date: November 1999
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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 65 Years   (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
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
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
Study Chair: John Barranger University of Pittsburgh
  More Information

ClinicalTrials.gov Identifier: NCT00004293     History of Changes
Other Study ID Numbers: 199/11725
UPITTS-M1230
First Submitted: October 18, 1999
First Posted: October 19, 1999
Last Update Posted: December 9, 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:
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