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Trial record 1 of 1 for:    NCT00358943
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International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

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ClinicalTrials.gov Identifier: NCT00358943
Recruitment Status : Recruiting
First Posted : August 1, 2006
Last Update Posted : January 13, 2023
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief 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.


Condition or disease
Gaucher Disease Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease

Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 12000 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 12 Months
Official Title: Gaucher Disease Registry Protocol
Actual Study Start Date : April 1991
Estimated Primary Completion Date : January 31, 2034
Estimated Study Completion Date : January 31, 2034

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort
Patients in ICGG Gaucher Registry
No experimental intervention is given. A patient with Gaucher Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.
Pregnant women with confirmed diagnosis of Gaucher disease
No experimental intervention is given. Pregnant women with confirmed diagnosis of Gaucher disease who are participating in the ICGG Gaucher Registry and consented to participate in the Gaucher Pregnancy Sub-registry, regardless of whether she is receiving disease-specific therapy and irrespective of the commercial product with which she may be treated.


Outcome Measures
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Primary Outcome Measures :
  1. ICGG Gaucher Registry: 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: 42 years ]
  2. Gaucher Pregnancy Sub-registry: 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, such as ERT with imiglucerase [ Time Frame: 42 years ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the ICGG Gaucher Registry.

For Gaucher Pregnancy Sub-registry: It is recommended that pregnancy data be collected on all eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, data collection for each individual pregnancy is encouraged for all women enrolled in this Sub-registry.

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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00358943


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com
Contact: ICGG Gaucher Registry HelpLine +1(617) 252-7500

Locations
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Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Study Director Genzyme, a Sanofi Company
More Information
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Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00358943    
Other Study ID Numbers: DIREGC07009
ICGG Gaucher Registry ( Other Identifier: Sanofi )
First Posted: August 1, 2006    Key Record Dates
Last Update Posted: January 13, 2023
Last Verified: January 12, 2023
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
Gaucher Disease
Glucocerebrosidase Deficiency Disease
Additional relevant MeSH terms:
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Gaucher Disease
Lipidoses
Deficiency Diseases
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
Malnutrition
Nutrition Disorders