International Collaborative Gaucher Group (ICGG) Gaucher Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2016 by Sanofi
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company ) Identifier:
First received: July 12, 2006
Last updated: February 18, 2016
Last verified: February 2016

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.

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:

Further study details as provided by Sanofi:

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)

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

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00358943

Contact: For site information, send an email with site number to
Contact: ICGG Gaucher Registry HelpLine +1 617-591-5500

  Show 63 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Sanofi ( Genzyme, a Sanofi Company ) Identifier: NCT00358943     History of Changes
Other Study ID Numbers: ICGG Gaucher Registry  DIREGC07009 
Study First Received: July 12, 2006
Last Updated: February 18, 2016
Health Authority: United States: Institutional Review Board

Keywords provided by Sanofi:
Gaucher Disease
Glucocerebrosidase Deficiency Disease

Additional relevant MeSH terms:
Deficiency Diseases
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
Nervous System Diseases
Nutrition Disorders
Metabolism, Inborn Errors
Sphingolipidoses processed this record on May 24, 2016