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

Gaucher Disease Registry Protocol

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: 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 willing to sign a consent form and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed. Who Should NOT Join This Trial: \- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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 (20)

Phoenix Children's Hospital- Site Number : 840003
Phoenix, Arizona, United States
University of Arizona- Site Number : 840015
Tucson, Arizona, United States
Arkansas Childrens Hospital- Site Number : 840109
Little Rock, Arkansas, United States
University of Arkansas for Medical Sciences- Site Number : 840113
Little Rock, Arkansas, United States
Tower Hematology/Oncology Medical Group- Site Number : 840056
Beverly Hills, California, United States
University of California at Irvine- Site Number : 840036
Irvine, California, United States
Southern California Permanente Medical Group- Site Number : 840108
Los Angeles, California, United States
USC Health Sciences Center Dept of Genetics- Site Number : 840082
Los Angeles, California, United States
Children's Hospital of Orange County- Site Number : 840074
Orange, California, United States
Stanford Unviersity MC Dept of Genetics- Site Number : 840022
Palo Alto, California, United States
UC Davis MIND Institute- Site Number : 840010
Sacramento, California, United States
Kaiser Permanente Hospital- Site Number : 840042
Sacramento, California, United States
University of California at San Diego- Site Number : 840007
San Diego, California, United States
University of California at San Francisco- Site Number : 840051
San Francisco, California, United States
Yale - Site Number : 840047
New Haven, Connecticut, United States
Children's National Medical Center- Site Number : 840067
Washington D.C., District of Columbia, United States
University Hematology Oncology- Site Number : 840075
Coral Springs, Florida, United States
University of Florida Pediatrics Genetics- Site Number : 840121
Jacksonville, Florida, United States
The Atwal Clinic- Site Number : 840112
Jacksonville, Florida, United States
University Of Miami SOM- Site Number : 840006
Miami, Florida, United States