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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT00358943
Ethics application status
Date submitted
12/07/2006
Date registered
1/08/2006
Date last updated
10/06/2019

Titles & IDs
Public title
International Collaborative Gaucher Group (ICGG) Gaucher Registry
Scientific title
International Collaborative Gaucher Group (ICGG) Gaucher Registry
Secondary ID [1] 0 0
DIREGC07009
Secondary ID [2] 0 0
ICGG Gaucher Registry
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
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

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

Outcomes
Primary outcome [1] 0 0
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
For duration of the study

Eligibility
Key inclusion criteria
- 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.
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- No exclusion criteria for participation in the ICGG Gaucher Registry.NOTE: Registry
participation does not exclude participation in other clinical studies.

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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigational Site Number 151002 - Adelaide
Recruitment hospital [2] 0 0
Investigational Site Number 151431 - Camperdown
Recruitment hospital [3] 0 0
Investigational Site Number 151011 - Clayton
Recruitment hospital [4] 0 0
Investigational Site Number 151491 - Douglas
Recruitment hospital [5] 0 0
Investigational Site Number 151725 - Hobart
Recruitment hospital [6] 0 0
Investigational Site Number 151799 - Nambour
Recruitment hospital [7] 0 0
Investigational Site Number 151008 - Parkville
Recruitment hospital [8] 0 0
Investigational Site Number 151069 - Westmead
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
2050 - Camperdown
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
4814 - Douglas
Recruitment postcode(s) [5] 0 0
7000 - Hobart
Recruitment postcode(s) [6] 0 0
4560 - Nambour
Recruitment postcode(s) [7] 0 0
3050 - Parkville
Recruitment postcode(s) [8] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
State/province [2] 0 0
Arkansas
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United States of America
State/province [3] 0 0
California
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United States of America
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Connecticut
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District of Columbia
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Florida
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Georgia
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United States of America
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Illinois
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United States of America
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Indiana
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United States of America
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Iowa
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United States of America
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Kentucky
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United States of America
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Louisiana
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United States of America
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Maine
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United States of America
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Maryland
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United States of America
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Massachusetts
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United States of America
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Michigan
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United States of America
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Minnesota
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United States of America
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Missouri
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United States of America
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Montana
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United States of America
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Nebraska
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United States of America
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New Jersey
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United States of America
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New York
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United States of America
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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United States of America
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Rhode Island
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United States of America
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South Carolina
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United States of America
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Tennessee
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United States of America
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Virginia
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United States of America
State/province [31] 0 0
Washington
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United States of America
State/province [32] 0 0
Wisconsin
Country [33] 0 0
Argentina
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Buenos Aires
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Argentina
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Caba
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Argentina
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Corrientes
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Argentina
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Córdoba
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Argentina
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La Plata
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Argentina
State/province [38] 0 0
Sarandi
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Belgium
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Antwerpen
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Belgium
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Brugge
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Belgium
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Brussel
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Belgium
State/province [42] 0 0
Bruxelles
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Belgium
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Gent
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Belgium
State/province [44] 0 0
Leuven
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Brazil
State/province [45] 0 0
Campinas
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Brazil
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Curitiba
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Brazil
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Porto Alegre
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Brazil
State/province [48] 0 0
Ribeirão Preto
Country [49] 0 0
Brazil
State/province [49] 0 0
Rio de Janeiro
Country [50] 0 0
Brazil
State/province [50] 0 0
São Paulo
Country [51] 0 0
Canada
State/province [51] 0 0
Halifax
Country [52] 0 0
Canada
State/province [52] 0 0
Moncton
Country [53] 0 0
China
State/province [53] 0 0
Beijing
Country [54] 0 0
Colombia
State/province [54] 0 0
Bogota
Country [55] 0 0
Denmark
State/province [55] 0 0
København Ø
Country [56] 0 0
Egypt
State/province [56] 0 0
Alexandria
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Egypt
State/province [57] 0 0
Ash Sharqia
Country [58] 0 0
Egypt
State/province [58] 0 0
Assuit
Country [59] 0 0
Egypt
State/province [59] 0 0
Azarita
Country [60] 0 0
Egypt
State/province [60] 0 0
Cairo
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France
State/province [61] 0 0
ANGERS Cedex 01
Country [62] 0 0
France
State/province [62] 0 0
Besancon
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France
State/province [63] 0 0
Bordeaux
Country [64] 0 0
France
State/province [64] 0 0
Brest
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France
State/province [65] 0 0
Caen
Country [66] 0 0
France
State/province [66] 0 0
CLICHY Cedex
Country [67] 0 0
France
State/province [67] 0 0
LYON Cedex 03
Country [68] 0 0
France
State/province [68] 0 0
MARSEILLE cdx 08
Country [69] 0 0
France
State/province [69] 0 0
Montpellier
Country [70] 0 0
France
State/province [70] 0 0
NANTES Cedex 1
Country [71] 0 0
France
State/province [71] 0 0
Paris
Country [72] 0 0
France
State/province [72] 0 0
Toulouse
Country [73] 0 0
France
State/province [73] 0 0
TOURS Cedex 9
Country [74] 0 0
Germany
State/province [74] 0 0
Gießen
Country [75] 0 0
Greece
State/province [75] 0 0
Athens
Country [76] 0 0
Greece
State/province [76] 0 0
Patras
Country [77] 0 0
Greece
State/province [77] 0 0
Thessaloniki
Country [78] 0 0
Hong Kong
State/province [78] 0 0
Kowloon
Country [79] 0 0
Hungary
State/province [79] 0 0
Létavértes
Country [80] 0 0
Hungary
State/province [80] 0 0
Nyíregyháza
Country [81] 0 0
Hungary
State/province [81] 0 0
Székesfehérvár
Country [82] 0 0
India
State/province [82] 0 0
Kolkata
Country [83] 0 0
Israel
State/province [83] 0 0
Haifa
Country [84] 0 0
Italy
State/province [84] 0 0
Catania
Country [85] 0 0
Italy
State/province [85] 0 0
Firenze
Country [86] 0 0
Italy
State/province [86] 0 0
Genova
Country [87] 0 0
Italy
State/province [87] 0 0
Milano
Country [88] 0 0
Italy
State/province [88] 0 0
Modena
Country [89] 0 0
Italy
State/province [89] 0 0
Monza (MB)
Country [90] 0 0
Italy
State/province [90] 0 0
Monza
Country [91] 0 0
Italy
State/province [91] 0 0
Napoli
Country [92] 0 0
Italy
State/province [92] 0 0
Padova
Country [93] 0 0
Italy
State/province [93] 0 0
Roma
Country [94] 0 0
Italy
State/province [94] 0 0
Udine
Country [95] 0 0
Japan
State/province [95] 0 0
Abeno-ku
Country [96] 0 0
Japan
State/province [96] 0 0
Chiba
Country [97] 0 0
Japan
State/province [97] 0 0
Setagaya
Country [98] 0 0
Japan
State/province [98] 0 0
Yonago-shi
Country [99] 0 0
Korea, Republic of
State/province [99] 0 0
Busan
Country [100] 0 0
Korea, Republic of
State/province [100] 0 0
Daejeon
Country [101] 0 0
Korea, Republic of
State/province [101] 0 0
Jeju-si
Country [102] 0 0
Korea, Republic of
State/province [102] 0 0
Jeonnam
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Korea, Republic of
State/province [103] 0 0
Seoul
Country [104] 0 0
Korea, Republic of
State/province [104] 0 0
Yangsan
Country [105] 0 0
Kuwait
State/province [105] 0 0
Kuwait
Country [106] 0 0
Lithuania
State/province [106] 0 0
Vilnius
Country [107] 0 0
Malaysia
State/province [107] 0 0
Jalan Pahang
Country [108] 0 0
Malaysia
State/province [108] 0 0
Kuala Lumpur
Country [109] 0 0
Netherlands
State/province [109] 0 0
Amsterdam
Country [110] 0 0
Peru
State/province [110] 0 0
Arequipa
Country [111] 0 0
Peru
State/province [111] 0 0
Callao
Country [112] 0 0
Philippines
State/province [112] 0 0
Manila
Country [113] 0 0
Poland
State/province [113] 0 0
Warszawa
Country [114] 0 0
Portugal
State/province [114] 0 0
Lisboa
Country [115] 0 0
Portugal
State/province [115] 0 0
Ponta Delgada
Country [116] 0 0
Russian Federation
State/province [116] 0 0
Moscow
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Russian Federation
State/province [117] 0 0
sait-Petersburg
Country [118] 0 0
Singapore
State/province [118] 0 0
Singapore
Country [119] 0 0
Slovakia
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Bratislava
Country [120] 0 0
South Africa
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Johannesburg
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South Africa
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Sandton
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Spain
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Barcelona
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Spain
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Córdoba
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Spain
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Madrid
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Spain
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Sabadell
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Spain
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Sevilla
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Spain
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Valencia
Country [128] 0 0
Spain
State/province [128] 0 0
Vigo
Country [129] 0 0
Spain
State/province [129] 0 0
Zaragoza
Country [130] 0 0
Sweden
State/province [130] 0 0
Borås
Country [131] 0 0
Sweden
State/province [131] 0 0
Malmö
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Sweden
State/province [132] 0 0
Norrköping
Country [133] 0 0
Taiwan
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Chiayi
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Taiwan
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Hualien
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Taiwan
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Kaohsiung Hsien,
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Taiwan
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Kaohsiung
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Taiwan
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New Taipei City
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Taiwan
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Putz City, Chia-yi
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Taiwan
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Taichung City
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Taiwan
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Taichung
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Taiwan
State/province [141] 0 0
Tainan
Country [142] 0 0
Taiwan
State/province [142] 0 0
Taipei
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Taiwan
State/province [143] 0 0
Tao Yuan County
Country [144] 0 0
Thailand
State/province [144] 0 0
Bangkok
Country [145] 0 0
Turkey
State/province [145] 0 0
Ankara
Country [146] 0 0
Turkey
State/province [146] 0 0
Istanbul
Country [147] 0 0
Turkey
State/province [147] 0 0
Malatya
Country [148] 0 0
United Arab Emirates
State/province [148] 0 0
Dubai
Country [149] 0 0
United Kingdom
State/province [149] 0 0
Birmingham

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 the understanding of the variability, progression, and natural history of
Gaucher disease with the ultimate goal of better guiding and assessing therapeutic
interventions;

- To provide the Gaucher medical community with recommendations for monitoring patients
and to provide reports on patient outcomes to help optimize patient care; and

- To evaluate the long-term effectiveness of Cerezyme.
Trial website
https://clinicaltrials.gov/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
Medical Monitor
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
For site information, send an email with site number to
Address 0 0
Country 0 0
Phone 0 0
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/show/NCT00358943