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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
17/04/2024
Titles & IDs
Public title
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
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Scientific title
Gaucher Disease Registry Protocol
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Secondary ID [1]
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ICGG Gaucher Registry
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Secondary ID [2]
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0
DIREGC07009
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease
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Cerebroside Lipidosis Syndrome
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0
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Glucocerebrosidase Deficiency Disease
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0
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Glucosylceramide Beta-Glucosidase Deficiency Disease
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0
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Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
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Other human genetics and inherited disorders
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Metabolic and Endocrine
0
0
0
0
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Metabolic disorders
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Metabolic and Endocrine
0
0
0
0
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Other metabolic disorders
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Diet and Nutrition
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0
0
0
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Other diet and nutrition disorders
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Intervention/exposure
Study type
Observational [Patient Registry]
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Patient registry
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Target follow-up duration
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Target follow-up type
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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.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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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
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Assessment method [1]
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Timepoint [1]
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42 years
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Primary outcome [2]
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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
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Assessment method [2]
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0
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Timepoint [2]
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42 years
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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.
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/04/1991
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
31/01/2034
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Actual
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Sample size
Target
12000
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,VIC,WA
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Recruitment hospital [1]
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Investigational Site Number : 151069 - Westmead
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Investigational Site Number : 151491 - Douglas
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Investigational Site Number : 151010 - Herston
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Investigational Site Number : 151002 - Adelaide
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Investigational Site Number : 151725 - Hobart
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Investigational Site Number : 151011 - Clayton
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Investigational Site Number : 151008 - Parkville
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Investigational Site Number : 151015 - Murdoch
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2145 - Westmead
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4814 - Douglas
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4006 - Herston
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5000 - Adelaide
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7000 - Hobart
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3168 - Clayton
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3050 - Parkville
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6150 - Murdoch
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Vietnam
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Ho Chi Minh City
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Genzyme, a Sanofi Company
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT00358943
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Trial related presentations / publications
El-Beshlawy A, Tylki-Szymanska A, Vellodi A, Belmatoug N, Grabowski GA, Kolodny EH, Batista JL, Cox GF, Mistry PK. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry. Mol Genet Metab. 2017 Jan-Feb;120(1-2):47-56. doi: 10.1016/j.ymgme.2016.12.001. Epub 2016 Dec 6. Mistry PK, Batista JL, Andersson HC, Balwani M, Burrow TA, Charrow J, Kaplan P, Khan A, Kishnani PS, Kolodny EH, Rosenbloom B, Scott CR, Weinreb N. Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry. Am J Hematol. 2017 Sep;92(9):929-939. doi: 10.1002/ajh.24801. Epub 2017 Jul 7. Weinreb NJ, Goldblatt J, Villalobos J, Charrow J, Cole JA, Kerstenetzky M, vom Dahl S, Hollak C. Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment. J Inherit Metab Dis. 2013 May;36(3):543-53. doi: 10.1007/s10545-012-9528-4. Epub 2012 Sep 14. Erratum In: J Inherit Metab Dis. 2014 Jan;37(1):147. Rosenbloom BE, Cappellini MD, Weinreb NJ, Dragosky M, Revel-Vilk S, Batista JL, Sekulic D, Mistry PK. Cancer risk and gammopathies in 2123 adults with Gaucher disease type 1 in the International Gaucher Group Gaucher Registry. Am J Hematol. 2022 Oct;97(10):1337-1347. doi: 10.1002/ajh.26675. Epub 2022 Aug 24. Weinreb NJ, Camelo JS Jr, Charrow J, McClain MR, Mistry P, Belmatoug N; International Collaborative Gaucher Group (ICGG) Gaucher Registry (NCT00358943) investigators. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment. Mol Genet Metab. 2021 Feb;132(2):100-111. doi: 10.1016/j.ymgme.2020.12.295. Epub 2021 Jan 8. Weinreb NJ, Kaplan P. The history and accomplishments of the ICGG Gaucher registry. Am J Hematol. 2015 Jul;90 Suppl 1:S2-5. doi: 10.1002/ajh.24054. No abstract available. Buyers RP, Collin-Histed T. Foreword. Am J Hematol. 2015 Jul;90 Suppl 1:S1. doi: 10.1002/ajh.24053. No abstract available.
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Public notes
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Contacts
Principal investigator
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Study Director
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Genzyme, a Sanofi Company
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Contact person for public queries
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Trial Transparency email recommended (Toll free number for US & Canada)
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800-633-1610
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[email protected]
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Contact person for scientific queries
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What supporting documents are/will be available?
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https://clinicaltrials.gov/study/NCT00358943
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