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Trial record 5 of 62 for:    "Gaucher disease"
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International Collaborative Gaucher Group (ICGG) Gaucher Registry

This study is currently recruiting participants.
Verified March 2013 by Genzyme
Sponsor:
Information provided by (Responsible Party):
Genzyme
ClinicalTrials.gov Identifier:
NCT00358943
First received: July 12, 2006
Last updated: March 12, 2013
Last verified: March 2013
History of Changes
  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 Cerezyme.

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:

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:
  • To provide the Gaucher medical community with recommendations for monitoring patients and to provide reports on patient outcomes to help optimize patient care. [ Time Frame: For duration of the study ] [ Designated as safety issue: No ]

Estimated Enrollment: 12000
Study Start Date: April 1991
Estimated Study Completion Date: January 2030
Estimated Primary Completion Date: January 2030 (Final data collection date for primary outcome measure)
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 - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com
  • 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: March 12, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Genzyme:
Gaucher Disease
Glucocerebrosidase Deficiency Disease

Additional relevant MeSH terms:
Gaucher Disease
Deficiency Diseases
Lipidoses
Malnutrition
Nutrition Disorders
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on May 22, 2013