International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT00358943 |
|
Recruitment Status :
Recruiting
First Posted : August 1, 2006
Last Update Posted : July 17, 2020
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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 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 |
| 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.
|
- 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 ]
- 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 ]
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, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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.
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.
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
| 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 |
Show 221 study locations
| Study Director: | Study Director | Genzyme, a Sanofi Company |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| 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: | July 17, 2020 |
| Last Verified: | July 2020 |
|
Gaucher Disease Glucocerebrosidase Deficiency Disease |
|
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 |