Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

This study is currently recruiting participants.

Verified by National Center for Research Resources (NCRR), December 2003

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

Sponsors and Collaborators:	National Center for Research Resources (NCRR) 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

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher's Disease

Drug Information available for: Alglucerase Imiglucerase

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment

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

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

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Lysosomal Storage Diseases
Rare Diseases
Sphingolipidosis
Central Nervous System Diseases
Brain Diseases
Metabolism, Inborn Errors

Lymphatic Diseases
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Gaucher Disease
Metabolic Disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Reticuloendotheliosis
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Brain Diseases

Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Gaucher Disease
Lipid Metabolism Disorders
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on May 07, 2009