Summary

Location
at San Francisco, California and other locations
Dates
study started
estimated completion

Description

Summary

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.

Official Title

Gaucher Disease Registry Protocol

Keywords

Gaucher Disease Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Lipidoses Deficiency Diseases

Eligibility

You can join if…

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.

You CAN'T join if...

  • No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.

Locations

  • University of California at San Francisco-Site Number:840051 accepting new patients
    San Francisco California United States
  • Stanford Unviersity MC Dept of Genetics-Site Number:840022 accepting new patients
    Palo Alto California 94305 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Genzyme, a Sanofi Company
ID
NCT00358943
Study Type
Observational [Patient Registry]
Participants
Expecting 12000 study participants
Last Updated