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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/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
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 : 151069 - Westmead
Recruitment hospital [2] 0 0
Investigational Site Number : 151491 - Douglas
Recruitment hospital [3] 0 0
Investigational Site Number : 151010 - Herston
Recruitment hospital [4] 0 0
Investigational Site Number : 151002 - Adelaide
Recruitment hospital [5] 0 0
Investigational Site Number : 151725 - Hobart
Recruitment hospital [6] 0 0
Investigational Site Number : 151011 - Clayton
Recruitment hospital [7] 0 0
Investigational Site Number : 151008 - Parkville
Recruitment hospital [8] 0 0
Investigational Site Number : 151015 - Murdoch
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4814 - Douglas
Recruitment postcode(s) [3] 0 0
4006 - Herston
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
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7000 - Hobart
Recruitment postcode(s) [6] 0 0
3168 - Clayton
Recruitment postcode(s) [7] 0 0
3050 - Parkville
Recruitment postcode(s) [8] 0 0
6150 - Murdoch
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00358943
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.
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00358943