International Collaborative Gaucher Group (ICGG) Gaucher Registry - Full Text View

Sponsor:	Genzyme
Information provided by (Responsible Party):	Genzyme
ClinicalTrials.gov Identifier:	NCT00358943

Purpose

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

The objectives of the Registry are:

To enhance the understanding of the variability, progression, and natural history of Gaucher disease with the ultimate goal of better guiding and assessing therapeutic interventions;
To provide the Gaucher medical community with recommendations for monitoring patients and to provide reports on patient outcomes to help optimize patient care; and
To evaluate the long-term effectiveness of enzyme replacement therapy (ERT).

Condition
Gaucher Disease Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease

Study Type:	Observational
Study Design:	Observational Model: Cohort Time Perspective: Prospective
Official Title:	International Collaborative Gaucher Group (ICGG) Gaucher Registry

Resource links provided by NLM:

Genetics Home Reference related topics: argininosuccinic aciduria ataxia neuropathy spectrum carbamoyl phosphate synthetase I deficiency Chanarin-Dorfman syndrome childhood myocerebrohepatopathy spectrum cholesteryl ester storage disease citrullinemia deoxyguanosine kinase deficiency Farber lipogranulomatosis Gaucher disease mitochondrial neurogastrointestinal encephalopathy disease myoclonic epilepsy myopathy sensory ataxia N-acetylglutamate synthase deficiency ornithine translocase deficiency Schindler disease succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher's Disease

U.S. FDA Resources

Further study details as provided by Genzyme:

Study Start Date:

April 1991

Detailed Description:

The ICGG Gaucher Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

In Asia-Pacific - Margie Jimenez, +852 2810 1613
In Europe - +31-35-699-1232, europe@gaucherregistry.com
In Latin America - +617-591-5500, help@gaucherregistry.com
In North America - +617-591-5500, help@gaucherregistry.com

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Gaucher disease

Criteria

Inclusion Criteria:

All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the Registry. Confirmed diagnosis is defined as a documented β-glucocerebrosidase deficiency and/or mutation in the β-glucocerebrosidase gene.
For all patients, appropriate patient authorization will be obtained.

Exclusion Criteria:

No exclusion criteria for participation in the ICGG Gaucher Registry.NOTE: Registry participation does not exclude participation in other clinical studies.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00358943

Contacts

Contact: ICGG Gaucher Registry HelpLine	800-745-4447 ext x15500	help@gaucherregistry.com
Contact: ICGG Gaucher Registry HelpLine	617-591-5500	help@gaucherregistry.com

Locations

United States, Massachusetts
Registry participation is worldwide and not limited to this facility; facilities not yet active may enroll upon identification of a patient.	Recruiting
Cambridge, Massachusetts, United States, 02142

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Medical Monitor

Genzyme

More Information

No publications provided

Responsible Party:	Genzyme
ClinicalTrials.gov Identifier:	NCT00358943 History of Changes
Other Study ID Numbers:	ICGG Gaucher Registry
Study First Received:	July 12, 2006
Last Updated:	December 8, 2011
Health Authority:	United States: Institutional Review Board

Keywords provided by Genzyme:

Gaucher Disease
Glucocerebrosidase Deficiency Disease

Additional relevant MeSH terms:

Deficiency Diseases
Carbamoyl-Phosphate Synthase I Deficiency Disease
Gaucher Disease
Lipidoses
Malnutrition
Nutrition Disorders
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases

Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Mitochondrial Diseases
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on February 09, 2012