Clinical trial NCT00358943: International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Official title: Gaucher Disease Registry Protocol
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Gaucher Disease
Cerebroside Lipidosis Syndrome
Glucocerebrosidase Deficiency Disease
Glucosylceramide Beta-Glucosidase Deficiency Disease
Keywords: Gaucher Disease
Glucocerebrosidase Deficiency Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2006-08-01
Enrollment: 12000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 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.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: 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
timeFrame: 42 years
measure: 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
timeFrame: 42 years
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital- Site Number : 840003
state: Arizona
city: Tucson
country: United States
facility: University of Arizona- Site Number : 840015
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Childrens Hospital- Site Number : 840109
state: Arkansas
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences- Site Number : 840113
state: Arkansas
city: Beverly Hills
country: United States
facility: Tower Hematology/Oncology Medical Group- Site Number : 840056
state: California
city: Irvine
country: United States
facility: University of California at Irvine- Site Number : 840036
state: California
city: Los Angeles
country: United States
facility: Southern California Permanente Medical Group- Site Number : 840108
state: California
city: Los Angeles
country: United States
facility: USC Health Sciences Center Dept of Genetics- Site Number : 840082
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County- Site Number : 840074
state: California
city: Palo Alto
country: United States
facility: Stanford Unviersity MC Dept of Genetics- Site Number : 840022
state: California
city: Sacramento
country: United States
facility: UC Davis MIND Institute- Site Number : 840010
state: California
city: Sacramento
country: United States
facility: Kaiser Permanente Hospital- Site Number : 840042
state: California
city: San Bernardino
country: United States
facility: Loma Linda University Children's Hospital- Site Number : 840117
state: California
city: San Diego
country: United States
facility: University of California at San Diego- Site Number : 840007
state: California
city: San Francisco
country: United States
facility: University of California at San Francisco- Site Number : 840051
state: California
city: New Haven
country: United States
facility: Yale - Site Number : 840047
state: Connecticut
city: Washington
country: United States
facility: Children's National Medical Center- Site Number : 840067
state: District of Columbia
city: Coral Springs
country: United States
facility: University Hematology Oncology- Site Number : 840075
state: Florida
city: Jacksonville
country: United States
facility: University of Florida Pediatrics Genetics- Site Number : 840121
state: Florida
city: Jacksonville
country: United States
facility: The Atwal Clinic- Site Number : 840112
state: Florida
city: Miami
country: United States
facility: University Of Miami SOM- Site Number : 840006
state: Florida
city: Tampa
country: United States
facility: University of Florida-Genetics- Site Number : 840096
state: Florida
city: Tampa
country: United States
facility: Pediatric Endocrine Associates- Site Number : 840017
state: Florida
city: Decatur
country: United States
facility: Emory University School of Medicine- Human Genetics- Site Number : 840060
state: Georgia
city: Aurora
country: United States
facility: University of Colorado at Denver Genetics- Site Number : 840068
state: Illinois
city: Chicago
country: United States
facility: Ann and Robert Lurie Children's Hospital- Site Number : 840011
state: Illinois
city: Chicago
country: United States
facility: Ann and Robert Lurie Children's Hospital- Site Number : 840013
state: Illinois
city: Chicago
country: United States
facility: Rush University Medical Center Genetics- Site Number : 840079
state: Illinois
city: Indianapolis
country: United States
facility: Indianapolis University School of Medicine- Site Number : 840027
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa- Site Number : 840032
state: Iowa
city: Louisville
country: United States
facility: University of Louisville- Genetics- Site Number : 840030
state: Kentucky
city: New Orleans
country: United States
facility: Tulane University Medical Center- Site Number : 840001
state: Louisiana
city: New Orleans
country: United States
facility: Ochsner Medical Center - Site Number : 840120
state: Louisiana
city: Portland
country: United States
facility: Maine Medical Center Pediatrics- Site Number : 840064
state: Maine
city: Baltimore
country: United States
facility: John Hopkins- Site Number : 840044
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital-Genetics- Site Number : 840062
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital- Site Number : 840092
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Pediatrics- Site Number : 840107
state: Michigan
city: Detroit
country: United States
facility: Children's Hospital of Michigan- Site Number : 840066
state: Michigan
city: Grand Rapids
country: United States
facility: Infusion Associates- Site Number : 840050
state: Michigan
city: Minneapolis
country: United States
facility: Children's Health Care- Site Number : 840114
state: Minnesota
city: Minneapolis
country: United States
facility: Children's Hospital and Clinics of Minnesota- Site Number : 840046
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota Medical Center Pediatrics- Site Number : 840076
state: Minnesota
city: Columibia
country: United States
facility: University of Missouri Health System Department of Genetics- Site Number : 840031
state: Missouri
city: Kansas City
country: United States
facility: Children's Mercy Hospital- Pediatrics- Site Number : 840063
state: Missouri
city: Saint Louis
country: United States
facility: Washington University of St. Louis- Site Number : 840100
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center- Pediatrics- Site Number : 840084
state: Nebraska
city: Las Vegas
country: United States
facility: Children's Specialty Center of Nevada- Site Number : 840008
state: Nevada
city: Camden
country: United States
facility: Cooper Health Center- Site Number : 840098
state: New Jersey
city: Hackensack
country: United States
facility: Joseph M. Sanzari Children's Hospital- Site Number : 840101
state: New Jersey
city: Morristown
country: United States
facility: Atlantic Health System- Site Number : 840099
state: New Jersey
city: New Brunswick
country: United States
facility: St. Peter's University Hospital- Site Number : 840016
state: New Jersey
city: Paterson
country: United States
facility: St. Joseph's Children's Hospital- Site Number : 840057
state: New Jersey
city: Manhasset
country: United States
facility: Northwell Health- Site Number : 840102
state: New York
city: New York
country: United States
facility: New York University School Of Medicine- Site Number : 840040
state: New York
city: New York
country: United States
facility: Metropolitan Hospital Center- Site Number : 840110
state: New York
city: New York
country: United States
facility: Mt. Sinai School of Medicine- Site Number : 840005
state: New York
city: New York
country: United States
facility: Site Number : 840026
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center SOM- Site Number : 840105
state: New York
city: Syracuse
country: United States
facility: State University of New York- Site Number : 840052
state: New York
city: Valhalla
country: United States
facility: New York Medical College- Site Number : 840039
state: New York
city: Charlotte
country: United States
facility: Carolinas Medical Center Hospital- Site Number : 840065
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center Genetics Dept- Site Number : 840037
state: North Carolina
city: Cleveland
country: United States
facility: UHCMC- Site Number : 840119
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation Pediatrics- Site Number : 840048
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital- Site Number : 840091
state: Ohio
city: Dublin
country: United States
facility: LSD Data Registry Site LLC- Site Number : 840094
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University- Site Number : 840080
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia- Site Number : 840034
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh- Site Number : 840023
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital Genetics- Site Number : 840053
state: Rhode Island
city: Greenville
country: United States
facility: Greenwood Genetcs- Site Number : 840055
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Hospital-Pediatrics- Site Number : 840049
state: Tennessee
city: Dallas
country: United States
facility: Baylor Research Center- Site Number : 840058
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Health Care System- Site Number : 840024
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Department of Medical Genetics- Site Number : 840043
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia School of Medicine-Pediatrics- Site Number : 840078
state: Virginia
city: Fairfax
country: United States
facility: O&O Alpan, LLC- Site Number : 840025
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of the Kings Daughters- Site Number : 840072
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital- Site Number : 840028
state: Washington
city: Seattle
country: United States
facility: University Of Washington Medical Center- Site Number : 840059
state: Washington
city: Tacoma
country: United States
facility: Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115
state: Washington
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
state: Wisconsin
city: Caba
country: Argentina
facility: Investigational Site Number : 150661
state: Buenos Aires
city: La Plata
country: Argentina
facility: Investigational Site Number : 032003
state: Buenos Aires
city: Sarandi
country: Argentina
facility: Investigational Site Number : 151085
state: Buenos Aires
city: Buenos Aires
country: Argentina
facility: Investigational Site Number : 032012
city: Corrientes
country: Argentina
facility: Investigational Site Number : 154176
city: Córdoba
country: Argentina
facility: Investigational Site Number : 032011
city: Sydney
country: Australia
facility: Investigational Site Number : 151431
state: New South Wales
city: Westmead
country: Australia
facility: Investigational Site Number : 151069
state: New South Wales
city: Brisbane
country: Australia
facility: Investigational Site Number : 151010
state: Queensland
city: Douglas
country: Australia
facility: Investigational Site Number : 151491
state: Queensland
city: Adelaide
country: Australia
facility: Investigational Site Number : 151002
state: South Australia
city: Hobart
country: Australia
facility: Investigational Site Number : 151725
state: Tasmania
city: Clayton
country: Australia
facility: Investigational Site Number : 151011
state: Victoria
city: Parkville
country: Australia
facility: Investigational Site Number : 151008
state: Victoria
city: Murdoch
country: Australia
facility: Investigational Site Number : 151015
state: Western Australia
city: Antwerp
country: Belgium
facility: Investigational Site Number : 150722
city: Bruges
country: Belgium
facility: Investigational Site Number : 151156
city: Brussels
country: Belgium
facility: Investigational Site Number : 150944
city: Brussels
country: Belgium
facility: Investigational Site Number : 152345
city: Brussels
country: Belgium
facility: Investigational Site Number : 150517
city: Bruxelles
country: Belgium
facility: Investigational Site Number : 151796
city: Edegem
country: Belgium
facility: Investigational Site Number : 154285
city: Gent
country: Belgium
facility: Investigational Site Number : 151134
city: Leuven
country: Belgium
facility: Investigational Site Number : 151668
city: Curitiba
country: Brazil
facility: Hospital das Clínicas da Universidade Federal do Paraná - HCUFPR- Site Number : 076010
state: Paraná
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre- Site Number : 076007
state: Rio Grande Do Sul
city: Campinas
country: Brazil
facility: Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076012
state: São Paulo
city: Ribeirão Preto
country: Brazil
facility: Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 076001
state: São Paulo
city: SÃ£O Paulo
country: Brazil
facility: Hospital das ClÃ-nicas da Faculdade de Medicina da USP- Site Number : 076008
state: São Paulo
city: Campinas
country: Brazil
facility: Faculdade De Medicina - Unicamp- Site Number : 076005
city: Rio de Janeiro
country: Brazil
facility: HEMORIO- Site Number : 076006
city: São Paulo
country: Brazil
facility: Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 076002
city: Sofia
country: Bulgaria
facility: Investigational Site Number : 100001
city: Sofia
country: Bulgaria
facility: Investigational Site Number : 151164
city: Calgary
country: Canada
facility: Investigational Site Number : 124014
state: Alberta
city: Edmonton
country: Canada
facility: Investigational Site Number : 124002
state: Alberta
city: Vancouver
country: Canada
facility: Investigational Site Number : 124010
state: British Columbia
city: Vancouver
country: Canada
facility: Investigational Site Number : 124011
state: British Columbia
city: Winnipeg
country: Canada
facility: Investigational Site Number : 124019
state: Manitoba
city: Moncton
country: Canada
facility: Investigational Site Number : 124022
state: New Brunswick
city: Kingston
country: Canada
facility: Investigational Site Number : 124024
state: Ontario
city: Kingston
country: Canada
facility: Investigational Site Number : 124005
state: Ontario
city: London
country: Canada
facility: Investigational Site Number : 124021
state: Ontario
city: Toronto
country: Canada
facility: Investigational Site Number : 124006
state: Ontario
city: Toronto
country: Canada
facility: Investigational Site Number : 124013
state: Ontario
city: Sherbrooke
country: Canada
facility: Investigational Site Number : 124001
state: Quebec
city: Beijing
country: China
facility: Investigational Site Number : 151787
city: Beijing
country: China
facility: Investigational Site Number : 156003
city: Guangzhou
country: China
facility: Investigational Site Number : 152357
city: Hangzhou
country: China
facility: Investigational Site Number : 152358
city: Hangzhou
country: China
facility: Investigational Site Number : 152359
city: Shanghai
country: China
facility: Investigational Site Number : 152356
city: Shanghai
country: China
facility: Investigational Site Number : 150899
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910001
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910002
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910003
city: Praha 2
country: Czechia
facility: Investigational Site Number : 150809
city: København Ø
country: Denmark
facility: Investigational Site Number : 151229
city: Santo Domingo
country: Dominican Republic
facility: Investigational Site Number : 2140001
city: Alexandria
country: Egypt
facility: Investigational Site Number : 5748
city: Ash Sharqia
country: Egypt
facility: Investigational Site Number : 9889
city: Assuit
country: Egypt
facility: Investigational Site Number : 9888
city: Cairo
country: Egypt
facility: Investigational Site Number : 9829
city: Cairo
country: Egypt
facility: Investigational Site Number : 8345
city: Cairo
country: Egypt
facility: Investigational Site Number : 9830
city: Angers
country: France
facility: Investigational Site Number : 250015
city: Besançon
country: France
facility: Investigational Site Number : 250004
city: Bordeaux
country: France
facility: Investigational Site Number : 250008
city: Brest
country: France
facility: Investigational Site Number : 250005
city: BRON Cedex
country: France
facility: Investigational Site Number : 250014
city: Caen
country: France
facility: Investigational Site Number : 250002
city: CLICHY Cedex
country: France
facility: Investigational Site Number : 250001
city: Garches
country: France
facility: Investigational Site Number : 250003
city: LYON Cedex 03
country: France
facility: Investigational Site Number : 250009
city: Marseille
country: France
facility: Investigational Site Number : 250011
city: Montpellier
country: France
facility: Investigational Site Number : 2500013
city: NANTES Cedex 1
country: France
facility: Investigational Site Number : 250006
city: Paris
country: France
facility: Investigational Site Number : 250007
city: Paris
country: France
facility: Investigational Site Number : 250010
city: Rennes
country: France
facility: Investigational Site Number : 250012
city: Strasbourg
country: France
facility: Investigational Site Number : 250018
city: Toulouse
country: France
facility: Investigational Site Number : 250017
city: TOURS Cedex 9
country: France
facility: Investigational Site Number : 250016
city: Berlin
country: Germany
facility: Investigational Site Number : 151686
city: Düsseldorf
country: Germany
facility: Investigational Site Number : 150783
city: Gießen
country: Germany
facility: Investigational Site Number : 154150
city: Hamburg
country: Germany
facility: Investigational Site Number : 151369
city: Hamburg
country: Germany
facility: Investigational Site Number : 151368
city: Mainz
country: Germany
facility: Investigational Site Number : 150031
city: Oberhausen
country: Germany
facility: Investigational Site Number : 150432
city: Athens
country: Greece
facility: Investigational Site Number : 300002
city: Athens
country: Greece
facility: Investigational Site Number : 300004
city: Patras
country: Greece
facility: Investigational Site Number : 300003
city: Thessaloniki
country: Greece
facility: Investigational Site Number : 300001
city: Kowloon
country: Hong Kong
facility: Investigational Site Number : 344001
city: Budapest
country: Hungary
facility: Investigational Site Number : 3480006
city: Létavértes
country: Hungary
facility: Investigational Site Number : 3480003
city: Nyíregyháza
country: Hungary
facility: Investigational Site Number : 3480007
city: Székesfehérvár
country: Hungary
facility: Investigational Site Number : 3480008
city: Ahmedabad
country: India
facility: Investigational Site Number : 356003
city: Jaipur
country: India
facility: Investigational Site Number : 356004
city: Kolkata
country: India
facility: Investigational Site Number : 356001
city: Mumbai
country: India
facility: Investigational Site Number : 356005
city: Secunderabad
country: India
facility: Investigational Site Number : 3560006
city: Vellore
country: India
facility: Investigational Site Number : 356002
city: Jakarta
country: Indonesia
facility: Investigational Site Number : 360001
city: Haifa
country: Israel
facility: Investigational Site Number : 3760003
city: Jerusalem
country: Israel
facility: Investigational Site Number : 3760001
city: Petach-Tikva
country: Israel
facility: Investigational Site Number : 3760002
city: Catania
country: Italy
facility: Investigational Site Number : 380008
city: Firenze
country: Italy
facility: Investigational Site Number : 380009
city: Genova
country: Italy
facility: Investigational Site Number : 380006
city: Milano
country: Italy
facility: Investigational Site Number : 380004
city: Modena
country: Italy
facility: Investigational Site Number : 380005
city: Monza (MB)
country: Italy
facility: Investigational Site Number : 380002
city: Monza
country: Italy
facility: Investigational Site Number : 380011
city: Napoli
country: Italy
facility: Investigational Site Number : 380010
city: Padova
country: Italy
facility: Investigational Site Number : 380003
city: Rome
country: Italy
facility: Investigational Site Number : 380007
city: Udine
country: Italy
facility: Investigational Site Number : 380001
city: Abeno-ku
country: Japan
facility: Investigational Site Number : 153205
state: Osaka
city: Yonago-shi
country: Japan
facility: Investigational Site Number : 153203
state: Tottori
city: Chiba
country: Japan
facility: Investigational Site Number : 153217
city: Tokyo
country: Japan
facility: Investigational Site Number : 153204
city: Amman
country: Jordan
facility: Investigational Site Number : 4000001
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 151149
state: Gyeonggi-do
city: Yangsan-si
country: Korea, Republic of
facility: Investigational Site Number : 153178
state: Gyeongsangnam-do
city: Yangsan
country: Korea, Republic of
facility: Investigational Site Number : 154148
state: Gyeongsangnam-do
city: Yangsan
country: Korea, Republic of
facility: Investigational Site Number : 154208
state: Gyeongsangnam-do
city: Daejeon
country: Korea, Republic of
facility: Investigational Site Number : 153191
city: Jeonnam
country: Korea, Republic of
facility: Investigational Site Number : 153225
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153180
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153196
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153192
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153212
city: Seoul
country: Korea, Republic of
facility: Investigational Site Number : 153222
city: Kuwait
country: Kuwait
facility: Investigational Site Number : 154225
city: Beirut
country: Lebanon
facility: Investigational Site Number : 4220002
city: Beirut
country: Lebanon
facility: Investigational Site Number : 4220004
city: Beirut
country: Lebanon
facility: Investigational Site Number : 422001
city: Douris
country: Lebanon
facility: Investigational Site Number : 422006
city: Vilnius
country: Lithuania
facility: Investigational Site Number : 154237
city: Jalan Pahang
country: Malaysia
facility: Investigational Site Number : 458001
city: Kuala Lumpur
country: Malaysia
facility: Investigational Site Number : 458002
city: Amsterdam
country: Netherlands
facility: Investigational Site Number : 150149
city: Amsterdam
country: Netherlands
facility: Investigational Site Number : 151171
city: Karachi
country: Pakistan
facility: Investigational Site Number : 154264
city: Lahore
country: Pakistan
facility: Investigational Site Number : 154266
city: Arequipa
country: Peru
facility: Investigational Site Number : 604001
city: Callao
country: Peru
facility: Investigational Site Number : 604002
city: Manila
country: Philippines
facility: Investigational Site Number : 608001
city: Warszawa
country: Poland
facility: Investigational Site Number : 150575
city: Coimbra
country: Portugal
facility: Investigational Site Number : 620003
city: Lisboa
country: Portugal
facility: Investigational Site Number : 620004
city: Ponta Delgada
country: Portugal
facility: Investigational Site Number : 620009
city: Cluj Napoca
country: Romania
facility: Investigational Site Number : 151188
city: Cluj Napoca
country: Romania
facility: Investigational Site Number : 642002
city: Cluj-Napoca
country: Romania
facility: Investigational Site Number : 642001
city: Moscow
country: Russian Federation
facility: Investigational Site Number : 643001
city: sait-Petersburg
country: Russian Federation
facility: Investigational Site Number : 643002
city: Al Mubarraz
country: Saudi Arabia
facility: Investigational Site Number : 682002
city: Jeddah
country: Saudi Arabia
facility: Investigational Site Number : 682003
city: Makkah
country: Saudi Arabia
facility: Investigational Site Number : 682004
city: Belgrade
country: Serbia
facility: Investigational Site Number : 688001
city: Belgrade
country: Serbia
facility: Investigational Site Number : 688002
city: Belgrade
country: Serbia
facility: Investigational Site Number : 688003
city: Belgrade
country: Serbia
facility: Investigational Site Number : 688005
city: NIŠ
country: Serbia
facility: Investigational Site Number : 688004
city: Singapore
country: Singapore
facility: Investigational Site Number : 702001
city: Bratislava
country: Slovakia
facility: Investigational Site Number : 703001
city: Johannesburg
country: South Africa
facility: Investigational Site Number : 154138
city: Johannesburg
country: South Africa
facility: Investigational Site Number : 710-003
city: Sandton
country: South Africa
facility: Investigational Site Number : 150346
city: Alcorcón
country: Spain
facility: Investigational Site Number : 154119
state: Madrid
city: Seville
country: Spain
facility: Investigational Site Number : 153215
state: Sevilla
city: Seville
country: Spain
facility: Investigational Site Number : 153219
state: Sevilla
city: A Coruña
country: Spain
facility: Investigational Site Number : 154146
city: Barcelona
country: Spain
facility: Investigational Site Number : 153237
city: Barcelona
country: Spain
facility: Investigational Site Number : 154147
city: Madrid
country: Spain
facility: Investigational Site Number : 154163
city: Madrid
country: Spain
facility: Investigational Site Number : 154236
city: Madrid
country: Spain
facility: Investigational Site Number : 151524
city: Madrid
country: Spain
facility: Investigational Site Number : 153251
city: Madrid
country: Spain
facility: Investigational Site Number : 154199
city: Madrid
country: Spain
facility: Investigational Site Number : 154160
city: Sabadell
country: Spain
facility: Investigational Site Number : 154145
city: Santiago de Compostela
country: Spain
facility: Investigational Site Number : 154222
city: Valencia
country: Spain
facility: Investigational Site Number : 154205
city: Vigo
country: Spain
facility: Investigational Site Number : 153213
city: Zaragoza
country: Spain
facility: Investigational Site Number : 154238
city: Zaragoza
country: Spain
facility: Investigational Site Number : 153216
city: Borås
country: Sweden
facility: Investigational Site Number : 154095
city: Malmö
country: Sweden
facility: Investigational Site Number : 151726
city: Norrköping
country: Sweden
facility: Investigational Site Number : 154092
city: Bern
country: Switzerland
facility: Investigational Site Number : 157045
city: Chiayi
country: Taiwan
facility: Investigational Site Number : 154093
city: Hualien
country: Taiwan
facility: Investigational Site Number : 152152
city: Kaohsiung City
country: Taiwan
facility: Investigational Site Number : 152246
city: Kaohsiung
country: Taiwan
facility: Investigational Site Number : 152244
city: New Taipei City
country: Taiwan
facility: Investigational Site Number : 151534
city: Putz City, Chia-yi
country: Taiwan
facility: Investigational Site Number : 151500
city: Taichung
country: Taiwan
facility: Investigational Site Number : 151181
city: Taichung
country: Taiwan
facility: Investigational Site Number : 151179
city: Tainan City
country: Taiwan
facility: Investigational Site Number : 153248
city: Tainan City
country: Taiwan
facility: Investigational Site Number : 152153
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 151182
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 152226
city: Taipei
country: Taiwan
facility: Investigational Site Number : 152321
city: Taoyuan City
country: Taiwan
facility: Investigational Site Number : 153158
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764003
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764004
city: Bangkok
country: Thailand
facility: Investigational Site Number : 764001
city: Khon Kaen
country: Thailand
facility: Investigational Site Number : 764005
city: Adana
country: Turkey
facility: Investigational Site Number : 792006
city: Ankara
country: Turkey
facility: Investigational Site Number : 792005
city: Ankara
country: Turkey
facility: Investigational Site Number : 792001
city: Ankara
country: Turkey
facility: Investigational Site Number : 792002
city: Istanbul
country: Turkey
facility: Investigational Site Number : 792004
city: Izmir
country: Turkey
facility: Investigational Site Number : 792007
city: Malatya
country: Turkey
facility: Investigational Site Number : 792003
city: Dubai
country: United Arab Emirates
facility: Investigational Site Number : 7840001
city: Cambridge
country: United Kingdom
facility: Investigational Site Number : 151340
state: Cambridgeshire
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 152287
city: Birmingham
country: United Kingdom
facility: Investigational Site Number : 154240
city: London
country: United Kingdom
facility: Investigational Site Number : 151646
city: London
country: United Kingdom
facility: Investigational Site Number : 151123
city: London
country: United Kingdom
facility: Investigational Site Number : 150303
city: Manchester
country: United Kingdom
facility: Investigational Site Number : 150455
city: Salford
country: United Kingdom
facility: Investigational Site Number : 152361
city: Hanoi
country: Vietnam
facility: Investigational Site Number : 154212
city: Ho Chi Minh City
country: Vietnam
facility: Investigational Site Number : 704002
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Study Director
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: Buyers RP, Collin-Histed T. Foreword. Am J Hematol. 2015 Jul;90 Suppl 1:S1. doi: 10.1002/ajh.24053. No abstract available.