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International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

NCT00358943

RecruitingN/A

Sponsor: Genzyme, a Sanofi Company

Conditions: Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease

Countries: United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China

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 understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Gaucher disease population. * To evaluate the long-term effectiveness of imiglucerase and of eliglustat. Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria
Inclusion Criteria:

ICGG Gaucher Registry

* 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.

Gaucher Pregnancy Sub-registry:

* be enrolled in the ICGG Gaucher Registry.
* be pregnant, or have been pregnant with appropriate medical documentation available.
* provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.

Exclusion Criteria:

\- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.
Locations (318)
  • Phoenix, Arizona, United States
  • Tucson, Arizona, United States
  • Little Rock, Arkansas, United States
  • Little Rock, Arkansas, United States
  • Beverly Hills, California, United States
  • Irvine, California, United States
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • Orange, California, United States
  • Sacramento, California, United States
  • Sacramento, California, United States
  • San Diego, California, United States
  • San Francisco, California, United States
  • Stanford, California, United States
  • New Haven, Connecticut, United States
  • Washington D.C., District of Columbia, United States
  • Washington D.C., District of Columbia, United States
  • Coral Springs, Florida, United States
  • Jacksonville, Florida, United States
  • Jacksonville, Florida, United States
  • Miami, Florida, United States
  • Tampa, Florida, United States
  • Tampa, Florida, United States
  • Decatur, Georgia, United States
  • Aurora, Illinois, United States
  • Chicago, Illinois, United States
  • Chicago, Illinois, United States
  • Chicago, Illinois, United States
  • Indianapolis, Indiana, United States
  • Iowa City, Iowa, United States
  • + 288 more on CT.gov