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Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

The recruitment status of this study is unknown because the information has not been verified recently.
Verified December 2003 by National Center for Research Resources (NCRR).
Recruitment status was  Recruiting
Sponsor:
Collaborator:
University of Pittsburgh
Information provided by:
National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:
NCT00004293
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: December 2003
History of Changes
  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:

U.S. FDA Resources

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

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

ClinicalTrials.gov Identifier: NCT00004293     History of Changes
Other Study ID Numbers: 199/11725, UPITTS-M1230
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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

ClinicalTrials.gov processed this record on May 22, 2013