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
University of Pittsburgh
Information provided by:
National Center for Research Resources (NCRR) Identifier:
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: December 2003


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:


Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.


Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


--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

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Please refer to this study by its identifier: NCT00004293

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
Study Chair: John Barranger University of Pittsburgh
  More Information

No publications provided 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

Additional relevant MeSH terms:
Gaucher Disease
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Sphingolipidoses processed this record on October 13, 2015