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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00358943




Registration number
NCT00358943
Ethics application status
Date submitted
12/07/2006
Date registered
1/08/2006
Date last updated
20/11/2024

Titles & IDs
Public title
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Scientific title
Gaucher Disease Registry Protocol
Secondary ID [1] 0 0
ICGG Gaucher Registry
Secondary ID [2] 0 0
DIREGC07009
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease 0 0
Cerebroside Lipidosis Syndrome 0 0
Glucocerebrosidase Deficiency Disease 0 0
Glucosylceramide Beta-Glucosidase Deficiency Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
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.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
42 years
Primary outcome [2] 0 0
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
Timepoint [2] 0 0
42 years

Eligibility
Key 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 ß-glucocerebrosidase deficiency and/or mutation in the ß-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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/1991
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/01/2034
Actual
Sample size
Target
12000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,VIC,WA
Recruitment hospital [1] 0 0
Investigational Site Number : 151431 - Sydney
Recruitment hospital [2] 0 0
Investigational Site Number : 151069 - Westmead
Recruitment hospital [3] 0 0
Investigational Site Number : 151010 - Brisbane
Recruitment hospital [4] 0 0
Investigational Site Number : 151491 - Douglas
Recruitment hospital [5] 0 0
Investigational Site Number : 151002 - Adelaide
Recruitment hospital [6] 0 0
Investigational Site Number : 151725 - Hobart
Recruitment hospital [7] 0 0
Investigational Site Number : 151011 - Clayton
Recruitment hospital [8] 0 0
Investigational Site Number : 151008 - Parkville
Recruitment hospital [9] 0 0
Investigational Site Number : 151015 - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Sydney
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4029 - Brisbane
Recruitment postcode(s) [4] 0 0
4814 - Douglas
Recruitment postcode(s) [5] 0 0
5000 - Adelaide
Recruitment postcode(s) [6] 0 0
7000 - Hobart
Recruitment postcode(s) [7] 0 0
3168 - Clayton
Recruitment postcode(s) [8] 0 0
3050 - Parkville
Recruitment postcode(s) [9] 0 0
6150 - Murdoch
Recruitment outside Australia
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United States of America
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Arizona
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Illinois
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Indiana
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Iowa
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Kentucky
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Louisiana
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Maine
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Missouri
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Nebraska
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Nevada
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New Jersey
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New York
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North Carolina
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Tennessee
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Ash Sharqia
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Assuit
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Cairo
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France
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France
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France
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Paris
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France
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Rennes
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France
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Strasbourg
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France
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France
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TOURS Cedex 9
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Berlin
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Germany
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Düsseldorf
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Germany
State/province [88] 0 0
Gießen
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Germany
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Hamburg
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Germany
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Mainz
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Germany
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Oberhausen
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Greece
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Athens
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Greece
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Patras
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Greece
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Thessaloniki
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Hong Kong
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Kowloon
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Hungary
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Budapest
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Hungary
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Létavértes
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Hungary
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Hungary
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India
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Ahmedabad
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Kolkata
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Mumbai
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Vellore
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Haifa
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Gyeonggi-do
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Coimbra
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Taoyuan City
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Thailand
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Bangkok
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Adana
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Ankara
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Istanbul
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Izmir
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Malatya
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United Arab Emirates
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Dubai
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United Kingdom
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Cambridgeshire
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United Kingdom
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Birmingham
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London
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Manchester
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Salford
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Hanoi
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Ho Chi Minh City

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Genzyme, a Sanofi Company
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free number for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
Contact-Us@sanofi.com
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT00358943