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

First Received on October 18, 1999. Last Updated on June 23, 2005 History of Changes

Sponsor:	National Center for Research Resources (NCRR)
Collaborator:	University of Pittsburgh
Information provided by:	National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:	NCT00004293

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 II

Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease Schindler disease succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher's Disease

Drug Information available for: Alglucerase Imiglucerase

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 February 09, 2012